Clinical Trial Results:
An Open-label, Baseline-controlled, Multicenter, Phase 3 Dose-titration Study Followed by a Fixed-dose Observation Period to Evaluate Efficacy, Safety and Pharmacokinetics of Mirabegron in Children and Adolescents From 3 to Less Than 18 Years of Age with Neurogenic Detrusor Overactivity (NDO) on Clean Intermittent Catheterization
Summary
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EudraCT number |
2015-002876-25 |
Trial protocol |
DK LT NO BE SK RO LV HR |
Global end of trial date |
06 May 2019
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Results information
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Results version number |
v3(current) |
This version publication date |
11 Oct 2022
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First version publication date |
14 Nov 2019
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Other versions |
v1 , v2 |
Version creation reason |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
178-CL-206A
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02751931 | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
Acronym: Crocodile Study | ||
Sponsors
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Sponsor organisation name |
Astellas Pharma Europe B.V.
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Sponsor organisation address |
Sylviusweg 62, Leiden, Netherlands, 2333 BE
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Public contact |
Clinical Trial Disclosure, Astellas Pharma Europe B.V., astellas.resultsdisclosure@astellas.com
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Scientific contact |
Clinical Trial Disclosure, Astellas Pharma Europe B.V., astellas.resultsdisclosure@astellas.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
Yes
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EMA paediatric investigation plan number(s) |
EMEA-000597-PIP03-15 | ||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
06 May 2019
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
06 May 2019
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The primary objective of this study was to evaluate the efficacy of mirabegron after multiple-dose administration in the pediatric population.
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Protection of trial subjects |
This clinical study was written, conducted and reported in accordance with the protocol, International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) Guidelines, and applicable local regulations, including the European Directive 2001/20/EC, on the protection of human rights, and with the ethical principles that have their origin in the Declaration of Helsinki. Astellas ensures that the use and disclosure of protected health information (PHI) obtained during a research study complies with the federal, national and/or regional legislation related to the privacy and protection of personal information.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
17 Jun 2016
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Australia: 1
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Country: Number of subjects enrolled |
Belgium: 3
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Country: Number of subjects enrolled |
Croatia: 7
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Country: Number of subjects enrolled |
Denmark: 2
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Country: Number of subjects enrolled |
Israel: 1
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Country: Number of subjects enrolled |
Jordan: 3
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Country: Number of subjects enrolled |
Latvia: 2
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Country: Number of subjects enrolled |
Lithuania: 7
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Country: Number of subjects enrolled |
Malaysia: 4
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Country: Number of subjects enrolled |
Mexico: 3
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Country: Number of subjects enrolled |
Norway: 5
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Country: Number of subjects enrolled |
Philippines: 10
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Country: Number of subjects enrolled |
Poland: 17
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Country: Number of subjects enrolled |
Romania: 6
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Country: Number of subjects enrolled |
Serbia: 4
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Country: Number of subjects enrolled |
Slovakia: 3
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Country: Number of subjects enrolled |
Korea, Republic of: 6
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Country: Number of subjects enrolled |
Taiwan: 1
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Country: Number of subjects enrolled |
Turkey: 6
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Worldwide total number of subjects |
91
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EEA total number of subjects |
52
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
56
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Adolescents (12-17 years) |
35
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
Pediatric participants consisting of male and female children from 3 to <12 and adolescents from 12 to <18 years of age, with a body weight of ≥11 kg, with NDO on clean intermittent catheterization (CIC) were enrolled in this study. | |||||||||||||||||||||
Pre-assignment
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Screening details |
Eligible participants who met inclusion and none of the exclusion criteria were enrolled. Participants who received oral drug to manage their NDO completed a 2 week washout period. A total of 113 pediatric patients were screened, 22 of whom were screening failures. | |||||||||||||||||||||
Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | |||||||||||||||||||||
Allocation method |
Non-randomised - controlled
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Blinding used |
Not blinded | |||||||||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Children (3 to < 12 Years) | |||||||||||||||||||||
Arm description |
Children age 3 to < 12 received an initial dose of 25 mg of mirabegron (pediatric equivalent dose [PED25]), orally once daily. Initial dose was up-titrated at weeks 2, 4 or 8 up to 50 mg of mirabegron (pediatric equivalent dose [PED50]). After week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). | |||||||||||||||||||||
Arm type |
Experimental | |||||||||||||||||||||
Investigational medicinal product name |
Mirabegron Tablets
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Investigational medicinal product code |
YM178
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Other name |
Myrbetriq, Betmiga
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Participants received initial dose of 25 mg of mirabegron PED25 orally once daily. At weeks 2, 4 or 8, participants were up-titrated to PED50 based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT. Participants with a body weight ≥35 kg received mirabegron tablets. At week 24, participants on mirabegron oral suspension could switch to tablets if the body weight became ≥35 kg. Participants who received mirabegron oral suspension could switch to mirabegron tablets for acceptability reasons after sponsor’s prior approval and on a case-by-case basis.
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Investigational medicinal product name |
Mirabegron Oral Suspension
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Investigational medicinal product code |
YM178
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Other name |
Myrbetriq, Betmiga
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Pharmaceutical forms |
Oral suspension
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Routes of administration |
Oral use
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Dosage and administration details |
Participants received initial dose of 25 mg of mirabegron PED25 orally once daily. At weeks 2, 4 or 8, participants were up-titrated to PED50 based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT. Participants with a body weight <35 kg received mirabegron oral suspension. For participants with a body weight ≥35 kg who did not want to or were unable to take tablets, the oral suspension could have been supplied. Participants who received mirabegron tablets could switch to mirabegron oral suspension for acceptability reasons after sponsor’s prior approval and on a case-by-case basis. Mirabegron extended-release granules were reconstituted with water to prepare a mirabegron oral suspension of 8 mg/mL. Administration was via an oral syringe with a sip of water afterwards.
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Arm title
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Adolescents (12 to < 18 Years) | |||||||||||||||||||||
Arm description |
Adolescents age 12 to < 18 received an initial dose of 25 mg of mirabegron (PED25), orally once daily. Initial dose was up-titrated at weeks 2, 4 or 8 up to 50 mg of mirabegron (PED50). After week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). | |||||||||||||||||||||
Arm type |
Experimental | |||||||||||||||||||||
Investigational medicinal product name |
Mirabegron Oral Suspension
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Investigational medicinal product code |
YM178
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Other name |
Myrbetriq, Betmiga
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Pharmaceutical forms |
Oral suspension
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Routes of administration |
Oral use
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Dosage and administration details |
Participants received initial dose of 25 mg of mirabegron PED25 orally once daily. At weeks 2, 4 or 8, participants were up-titrated to PED50 based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT. Participants with a body weight <35 kg received mirabegron oral suspension. For participants with a body weight ≥35 kg who did not want to or were unable to take tablets, the oral suspension could have been supplied. Participants who received mirabegron tablets could switch to mirabegron oral suspension for acceptability reasons after sponsor’s prior approval and on a case-by-case basis. Mirabegron extended-release granules were reconstituted with water to prepare a mirabegron oral suspension of 8 mg/mL. Administration was via an oral syringe with a sip of water afterwards.
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Investigational medicinal product name |
Mirabegron Tablets
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Investigational medicinal product code |
YM178
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Other name |
Myrbetriq, Betmiga
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Participants received initial dose of 25 mg of mirabegron PED25 orally once daily. At weeks 2, 4 or 8, participants were up-titrated to PED50 based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT. Participants with a body weight ≥35 kg received mirabegron tablets. At week 24, participants on mirabegron oral suspension could switch to tablets if the body weight became ≥35 kg. Participants who received mirabegron oral suspension could switch to mirabegron tablets for acceptability reasons after sponsor’s prior approval and on a case-by-case basis.
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Baseline characteristics reporting groups
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Reporting group title |
Children (3 to < 12 Years)
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Reporting group description |
Children age 3 to < 12 received an initial dose of 25 mg of mirabegron (pediatric equivalent dose [PED25]), orally once daily. Initial dose was up-titrated at weeks 2, 4 or 8 up to 50 mg of mirabegron (pediatric equivalent dose [PED50]). After week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Adolescents (12 to < 18 Years)
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Reporting group description |
Adolescents age 12 to < 18 received an initial dose of 25 mg of mirabegron (PED25), orally once daily. Initial dose was up-titrated at weeks 2, 4 or 8 up to 50 mg of mirabegron (PED50). After week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Children (3 to < 12 Years)
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Reporting group description |
Children age 3 to < 12 received an initial dose of 25 mg of mirabegron (pediatric equivalent dose [PED25]), orally once daily. Initial dose was up-titrated at weeks 2, 4 or 8 up to 50 mg of mirabegron (pediatric equivalent dose [PED50]). After week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). | ||
Reporting group title |
Adolescents (12 to < 18 Years)
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Reporting group description |
Adolescents age 12 to < 18 received an initial dose of 25 mg of mirabegron (PED25), orally once daily. Initial dose was up-titrated at weeks 2, 4 or 8 up to 50 mg of mirabegron (PED50). After week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). | ||
Subject analysis set title |
Children PED25 (PKAS)
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
Participants aged 3 to < 12 years who received pediatric equivalent dose of 25 mg at the time of PK sampling.
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Subject analysis set title |
Adolescents PED25 (PKAS)
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
Participants aged 12 to < 18 years who received pediatric equivalent dose of 25 mg at the time of PK sampling.
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Subject analysis set title |
Children PED50 (PKAS)
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
Participants aged 3 to < 12 years who received pediatric equivalent dose of 50 mg at the time of PK sampling.
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Subject analysis set title |
Adolescents PED50 (PKAS)
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
Participants aged 12 to < 18 years who received pediatric equivalent dose of 50 mg at the time of PK sampling.
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End point title |
Change From Baseline in Maximum Cystometric Capacity (MCC) at Week 24 | ||||||||||||
End point description |
Change from baseline in MCC was based on filling urodynamics, volume at the end of filling. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. The analysis population consisted of the full analysis set (FAS) which consisted of all participants who took ≥ 1 dose of study drug and provided both valid (as by the central reviewer’s assessment) nonmissing MCC measurements at baseline and at a postbaseline visit for the primary efficacy endpoint. Missing MCC observations at week 24 were imputed using last observation carried forward (LOCF).
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End point type |
Primary
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End point timeframe |
Baseline and week 24
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Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||
Statistical analysis description |
Change from baseline in MCC in children at week 24. The number of participants included in this analysis was 43.
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Comparison groups |
Adolescents (12 to < 18 Years) v Children (3 to < 12 Years)
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Number of subjects included in analysis |
68
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
< 0.001 [1] | ||||||||||||
Method |
Paired t-test | ||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||
Point estimate |
72.09
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Confidence interval |
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level |
95% | ||||||||||||
sides |
2-sided
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lower limit |
45.28 | ||||||||||||
upper limit |
98.89 | ||||||||||||
Notes [1] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
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Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||
Statistical analysis description |
Change from baseline in MCC in adolescents at week 24. The number of participants included in this analysis was 25.
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Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
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Number of subjects included in analysis |
68
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
< 0.001 [2] | ||||||||||||
Method |
Paired t-test | ||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||
Point estimate |
113.21
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Confidence interval |
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level |
95% | ||||||||||||
sides |
2-sided
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lower limit |
78.95 | ||||||||||||
upper limit |
147.47 | ||||||||||||
Notes [2] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
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End point title |
Change From Baseline in MCC at Week 4 | ||||||||||||
End point description |
Change from baseline in MCC was based on filling urodynamics (volume at the end of filling). During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure.
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End point type |
Secondary
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End point timeframe |
Baseline and week 4
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Statistical analysis title |
Change From Baseline at Week 4 - Children | ||||||||||||
Statistical analysis description |
Change from baseline in MCC in children at week 4. The number of participants included in this analysis was 41.
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Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
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Number of subjects included in analysis |
64
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
< 0.001 [3] | ||||||||||||
Method |
Paired t-test | ||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||
Point estimate |
41.36
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Confidence interval |
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level |
95% | ||||||||||||
sides |
2-sided
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lower limit |
18.75 | ||||||||||||
upper limit |
63.97 | ||||||||||||
Notes [3] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
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Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | ||||||||||||
Statistical analysis description |
Change from baseline in MCC in adolescents at week 4. The number of participants included in this analysis was 25.
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Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
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Number of subjects included in analysis |
64
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
< 0.001 [4] | ||||||||||||
Method |
Paired t-test | ||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||
Point estimate |
80.78
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Confidence interval |
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level |
95% | ||||||||||||
sides |
2-sided
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lower limit |
39.2 | ||||||||||||
upper limit |
122.36 | ||||||||||||
Notes [4] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
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End point title |
Change From Baseline in Bladder Compliance (ΔV/ΔP) | ||||||||||||||||||
End point description |
Change from baseline in bladder compliance (change in volume/change in pressure) was assessed by the independent central reviewers and reported as annotations on the urodynamic trace and in an external database. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. The analysis population consisted of the FAS. N is the number of participants with available data at each time point.
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End point type |
Secondary
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End point timeframe |
Baseline and weeks 4 and 24
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Statistical analysis title |
Change From Baseline at Week 4 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in ΔV/ΔP in children at week 4. The number of participants included in this analysis was 39.
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Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
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Number of subjects included in analysis |
68
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||||||||
P-value |
= 0.618 [5] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-4.09
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Confidence interval |
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level |
95% | ||||||||||||||||||
sides |
2-sided
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lower limit |
-20.55 | ||||||||||||||||||
upper limit |
12.38 | ||||||||||||||||||
Notes [5] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
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Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in ΔV/ΔP in adolescents at week 4. The number of participants included in this analysis was 22.
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Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
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Number of subjects included in analysis |
68
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||||||||
P-value |
= 0.005 [6] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
15.16
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Confidence interval |
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level |
95% | ||||||||||||||||||
sides |
2-sided
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lower limit |
5.1 | ||||||||||||||||||
upper limit |
25.22 | ||||||||||||||||||
Notes [6] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
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Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in ΔV/ΔP in children at week 24. The number of participants included in this analysis was 33.
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Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
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Number of subjects included in analysis |
68
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Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.055 [7] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
14.62
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-0.31 | ||||||||||||||||||
upper limit |
29.54 | ||||||||||||||||||
Notes [7] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in ΔV/ΔP in adolescents at week 24. The number of participants included in this analysis was 21.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [8] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
13.59
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
6.75 | ||||||||||||||||||
upper limit |
20.42 | ||||||||||||||||||
Notes [8] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
|||||||||||||||||||
End point title |
Change From Baseline in Number of Overactive Detrusor Contractions (> 15 cm H20) Until End of Filling | ||||||||||||||||||
End point description |
Detrusor overactivity is the occurrence of involuntary detrusor contractions during filling cystometry. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. The analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
Baseline and weeks 4 and 24
|
||||||||||||||||||
|
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in number of overactive detrusor contractions in children at week 4. The number of participants included in this analysis was 41.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.632 [9] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
0.44
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-1.4 | ||||||||||||||||||
upper limit |
2.28 | ||||||||||||||||||
Notes [9] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in number of overactive detrusor contractions in children at week 24. The number of participants included in this analysis was 36.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.011 [10] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-1.86
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-3.27 | ||||||||||||||||||
upper limit |
-0.45 | ||||||||||||||||||
Notes [10] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in number of overactive detrusor contractions in adolescents at week 24. The number of participants included in this analysis was 22.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.359 [11] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-0.77
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-2.49 | ||||||||||||||||||
upper limit |
0.94 | ||||||||||||||||||
Notes [11] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in number of overactive detrusor contractionsin adolescents at week 4. The number of participants included in this analysis was 22.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.321 [12] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-0.64
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-1.94 | ||||||||||||||||||
upper limit |
0.67 | ||||||||||||||||||
Notes [12] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
|||||||||||||||||||
End point title |
Change From Baseline in Detrusor Pressure at End of Filling | ||||||||||||||||||
End point description |
Filling was stopped (end of filling) when the detrusor pressure exceeded 100 cm H2O or was considered dangerously high by the investigator or urodynamicist (for instance, a prolonged passive detrusor pressure > 40 cm H2O). During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. The analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
Baseline and weeks 4 and 24
|
||||||||||||||||||
|
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in detrusor pressure in children at week 4. The number of participants included in this analysis was 41.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [13] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-12.38
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-18.56 | ||||||||||||||||||
upper limit |
-6.21 | ||||||||||||||||||
Notes [13] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in detrusor pressure in children at week 24. The number of participants included in this analysis was 36.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [14] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-18.11
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-24.87 | ||||||||||||||||||
upper limit |
-11.35 | ||||||||||||||||||
Notes [14] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in detrusor pressure in adolescents at week 24. The number of participants included in this analysis was 22.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.005 [15] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-13.19
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-22.02 | ||||||||||||||||||
upper limit |
-4.36 | ||||||||||||||||||
Notes [15] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in detrusor pressure in adolescents at week 4. The number of participants included in this analysis was 22.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.334 [16] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-6.48
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-20.09 | ||||||||||||||||||
upper limit |
7.13 | ||||||||||||||||||
Notes [16] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Average Catheterized Volume Per Catheterization | |||||||||||||||||||||||||||||||||
End point description |
For each participant, the average catheterized volume per catheterization was calculated as the sum of all available/non-missing catheterized volumes recorded over 2 measuring days in the weekend diary, whether or not the 2 days were consecutive divided by the number of catheterizations with non-missing volumes. If volumes were recorded on 1 single day of the weekend diary, the average catheterized volume per catheterization was calculated using all available/non-missing catheterized volumes recorded that day. If no volumes were recorded on any day of the weekend diary, the average catheterized volume per catheterization was missing. A valid bladder diary day in the weekend diary was any e-diary day for which ≥1 catheterized volume >0 mL was recorded with complete date and time. Analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
|||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||
End point timeframe |
Baseline and weeks 2, 4, 8, 12, 24, 36, and 52
|
|||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in children at week 2. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.035 [17] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
14.58
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
1 | |||||||||||||||||||||||||||||||||
upper limit |
28.1 | |||||||||||||||||||||||||||||||||
Notes [17] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in adolescents at week 2. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.003 [18] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
35.99
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
13.1 | |||||||||||||||||||||||||||||||||
upper limit |
58.9 | |||||||||||||||||||||||||||||||||
Notes [18] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in children at week 4. The number of participants included in this analysis was 42.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [19] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
30.08
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
14.7 | |||||||||||||||||||||||||||||||||
upper limit |
45.5 | |||||||||||||||||||||||||||||||||
Notes [19] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in adolescents at week 4. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [20] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
51.96
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
24.6 | |||||||||||||||||||||||||||||||||
upper limit |
79.3 | |||||||||||||||||||||||||||||||||
Notes [20] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in children at week 8. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [21] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
36.9
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
22.7 | |||||||||||||||||||||||||||||||||
upper limit |
51.1 | |||||||||||||||||||||||||||||||||
Notes [21] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in adolescents at week 8. The number of participants included in this analysis was 2.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [22] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
45.1
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
21.3 | |||||||||||||||||||||||||||||||||
upper limit |
68.9 | |||||||||||||||||||||||||||||||||
Notes [22] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in children at week 12. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [23] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
32.25
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
18.2 | |||||||||||||||||||||||||||||||||
upper limit |
46.3 | |||||||||||||||||||||||||||||||||
Notes [23] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in adolescents at week 12. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.001 [24] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
43.94
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
19.2 | |||||||||||||||||||||||||||||||||
upper limit |
68.6 | |||||||||||||||||||||||||||||||||
Notes [24] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in children at week 24. The number of participants included in this analysis was 41.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [25] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
41.63
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
23.3 | |||||||||||||||||||||||||||||||||
upper limit |
60 | |||||||||||||||||||||||||||||||||
Notes [25] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in adolescents at week 24. The number of participants included in this analysis was 23.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.002 [26] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
59.31
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
23.8 | |||||||||||||||||||||||||||||||||
upper limit |
94.9 | |||||||||||||||||||||||||||||||||
Notes [26] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in children at week 36. The number of participants included in this analysis was 40.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [27] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
53.87
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
24.5 | |||||||||||||||||||||||||||||||||
upper limit |
83.2 | |||||||||||||||||||||||||||||||||
Notes [27] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in adolescents at week 36. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.002 [28] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
52.14
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
20.5 | |||||||||||||||||||||||||||||||||
upper limit |
83.8 | |||||||||||||||||||||||||||||||||
Notes [28] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in children at week 52. The number of participants included in this analysis was 40.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [29] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
42.84
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
22 | |||||||||||||||||||||||||||||||||
upper limit |
63.7 | |||||||||||||||||||||||||||||||||
Notes [29] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average catheterized volume per catheterization in adolescents at week 52. The number of participants included in this analysis was 23.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.008 [30] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
42.4
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
12.5 | |||||||||||||||||||||||||||||||||
upper limit |
72.3 | |||||||||||||||||||||||||||||||||
Notes [30] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Maximum Catheterized Volume | |||||||||||||||||||||||||||||||||
End point description |
For each participant, the maximum catheterized volume per day was calculated using all available/non-missing catheterized volumes recorded for the 2 measuring days in the weekend e-diary, whether or not these 2 days were consecutive. Maximum value was calculated separately for each measuring day & the mean of the two values was used. If volumes recorded on 1 single day of the weekend e-diary, the maximum catheterized volume per day was calculated using all available/non-zero catheterized volumes recorded that day. If no volumes were recorded on any day of the weekend e-diary, the maximum catheterized volume per day was missing. A valid bladder diary day in the weekend diary was any e-diary day for which ≥1 catheterized volume >0 mL was recorded with complete date and time. Analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
|||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||
End point timeframe |
Baseline and weeks 2, 4, 8, 12, 24, 36, and 52
|
|||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in children at week 2. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.126 [31] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
17.5
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-5.1 | |||||||||||||||||||||||||||||||||
upper limit |
40.1 | |||||||||||||||||||||||||||||||||
Notes [31] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in adolescents at week 2. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.014 [32] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
42.38
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
9.3 | |||||||||||||||||||||||||||||||||
upper limit |
75.4 | |||||||||||||||||||||||||||||||||
Notes [32] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in children at week 4. The number of participants included in this analysis was 42.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [33] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
46.69
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
21.7 | |||||||||||||||||||||||||||||||||
upper limit |
71.7 | |||||||||||||||||||||||||||||||||
Notes [33] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in adolescents at week 4. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.002 [34] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
73.25
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
29.3 | |||||||||||||||||||||||||||||||||
upper limit |
117.2 | |||||||||||||||||||||||||||||||||
Notes [34] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in children at week 8. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [35] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
45.27
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
22.1 | |||||||||||||||||||||||||||||||||
upper limit |
68.4 | |||||||||||||||||||||||||||||||||
Notes [35] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in adolescents at week 8. The number of participants included in this analysis was 22.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.02 | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
42.86
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
7.4 | |||||||||||||||||||||||||||||||||
upper limit |
78.3 | |||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in children at week 12. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.003 [36] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
33.23
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
12.2 | |||||||||||||||||||||||||||||||||
upper limit |
54.3 | |||||||||||||||||||||||||||||||||
Notes [36] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in children at week 24. The number of participants included in this analysis was 41.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.004 [37] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
49.88
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
17.1 | |||||||||||||||||||||||||||||||||
upper limit |
82.6 | |||||||||||||||||||||||||||||||||
Notes [37] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in adolescents at week 12. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.003 [38] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
47.29
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
17.8 | |||||||||||||||||||||||||||||||||
upper limit |
76.8 | |||||||||||||||||||||||||||||||||
Notes [38] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in adolescents at week 24. The number of participants included in this analysis was 23.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.003 [39] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
84.39
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
31.6 | |||||||||||||||||||||||||||||||||
upper limit |
137.1 | |||||||||||||||||||||||||||||||||
Notes [39] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in children at week 36. The number of participants included in this analysis was 40.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.003 [40] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
60.09
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
21.2 | |||||||||||||||||||||||||||||||||
upper limit |
99 | |||||||||||||||||||||||||||||||||
Notes [40] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in children at week 52. The number of participants included in this analysis was 40.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.001 [41] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
53.51
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
22.6 | |||||||||||||||||||||||||||||||||
upper limit |
84.4 | |||||||||||||||||||||||||||||||||
Notes [41] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in adolescents at week 36. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.017 [42] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
54.78
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
10.6 | |||||||||||||||||||||||||||||||||
upper limit |
98.9 | |||||||||||||||||||||||||||||||||
Notes [42] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized volume in adolescents at week 52. The number of participants included in this analysis was 23.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.021 [43] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
54.3
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
9 | |||||||||||||||||||||||||||||||||
upper limit |
99.6 | |||||||||||||||||||||||||||||||||
Notes [43] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Maximum Catheterized Daytime Volume (MCDV) | |||||||||||||||||||||||||||||||||
End point description |
For each participant, the MCDV was calculated using all available/non-missing catheterized daytime volumes for the 2 measuring days in the weekend e-diary, whether or not the 2 days were consecutive. Maximum value was calculated separately for each measuring day & the mean of the 2 values was used. If volumes were recorded on 1 single day of the weekend e-diary, the MCDV was calculated using all available/non-zero catheterized daytime volumes recorded that day. If no volumes were recorded on any day of the weekend e-diary, the MCDV was missing. Daytime was defined as the time between wake-up time (minus 30 min) & time to sleep (plus 29 min) recorded in the e-diary. A valid bladder diary day in the weekend diary was any e-diary day for which ≥1 catheterized volume >0 mL was recorded with complete date and time. Analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
|||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||
End point timeframe |
Baseline and weeks 2, 4, 8, 12, 24, 36, and 52
|
|||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in children at week 2. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.113 [44] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
18.13
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-4.5 | |||||||||||||||||||||||||||||||||
upper limit |
40.7 | |||||||||||||||||||||||||||||||||
Notes [44] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in adolescents at week 2. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.056 [45] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
35.58
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-1.1 | |||||||||||||||||||||||||||||||||
upper limit |
72.2 | |||||||||||||||||||||||||||||||||
Notes [45] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in adolescents at week 4. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.006 [46] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
70.35
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
22.2 | |||||||||||||||||||||||||||||||||
upper limit |
118.5 | |||||||||||||||||||||||||||||||||
Notes [46] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in children at week 4. The number of participants included in this analysis was 42.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.005 [47] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
37.71
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
11.7 | |||||||||||||||||||||||||||||||||
upper limit |
63.7 | |||||||||||||||||||||||||||||||||
Notes [47] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in adolescents at week 8. The number of participants included in this analysis was 22.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.063 [48] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
38.11
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-2.2 | |||||||||||||||||||||||||||||||||
upper limit |
78.4 | |||||||||||||||||||||||||||||||||
Notes [48] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in children at week 12. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Adolescents (12 to < 18 Years) v Children (3 to < 12 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.008 [49] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
29.05
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
8.2 | |||||||||||||||||||||||||||||||||
upper limit |
49.9 | |||||||||||||||||||||||||||||||||
Notes [49] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in children at week 8. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [50] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
43.91
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
21 | |||||||||||||||||||||||||||||||||
upper limit |
66.8 | |||||||||||||||||||||||||||||||||
Notes [50] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in children at week 24. The number of participants included in this analysis was 41.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.006 [51] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
44.2
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
13.2 | |||||||||||||||||||||||||||||||||
upper limit |
75.2 | |||||||||||||||||||||||||||||||||
Notes [51] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in adolescents at week 12. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.009 [52] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
43.04
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
11.9 | |||||||||||||||||||||||||||||||||
upper limit |
74.2 | |||||||||||||||||||||||||||||||||
Notes [52] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in adolescents at week 24. The number of participants included in this analysis was 23.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.003 [53] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
81.37
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
30.4 | |||||||||||||||||||||||||||||||||
upper limit |
132.3 | |||||||||||||||||||||||||||||||||
Notes [53] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in adolescents at week 36. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.039 [54] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
50.9
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
2.7 | |||||||||||||||||||||||||||||||||
upper limit |
99.1 | |||||||||||||||||||||||||||||||||
Notes [54] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in children at week 36. The number of participants included in this analysis was 40.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.004 [55] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
58.49
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
19.8 | |||||||||||||||||||||||||||||||||
upper limit |
97.2 | |||||||||||||||||||||||||||||||||
Notes [55] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in adolescents at week 52. The number of participants included in this analysis was 23.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.057 [56] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
49.13
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-1.6 | |||||||||||||||||||||||||||||||||
upper limit |
99.8 | |||||||||||||||||||||||||||||||||
Notes [56] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in maximum catheterized daytime volume in children at week 52. The number of participants included in this analysis was 40.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.002 [57] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
53.76
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
21.7 | |||||||||||||||||||||||||||||||||
upper limit |
85.8 | |||||||||||||||||||||||||||||||||
Notes [57] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Average Morning Catheterized Volume | |||||||||||||||||||||||||||||||||
End point description |
The first morning catheterized volume was the first recorded non-zero volume within or after the hour of the wake-up time on a volume-measuring day in the e-diary. The average first morning catheterized volume was calculated as the average of the available first morning catheterized volumes recorded for the 2 measuring days in the weekend e-diary, whether or not these 2 days were consecutive. If the first morning catheterized volume was recorded on 1 single day of the weekend e-diary, the average morning catheterized is the first morning catheterized that day. If no first morning catheterized volumes are recorded on any day of the weekend e-diary, the average first morning catheterized volume was missing. A valid bladder diary day in the weekend diary was any e-diary day for which ≥1 catheterized volume >0 mL was recorded with complete date and time. Analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
|||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||
End point timeframe |
Baseline and weeks 2, 4, 8, 12, 24, 36, and 52
|
|||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in adolescents at week 2. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.035 [58] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
39.52
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
3 | |||||||||||||||||||||||||||||||||
upper limit |
76 | |||||||||||||||||||||||||||||||||
Notes [58] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in children at week 2. The number of participants included in this analysis was 41.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.617 [59] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
7.98
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-24 | |||||||||||||||||||||||||||||||||
upper limit |
40 | |||||||||||||||||||||||||||||||||
Notes [59] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in children at week 4. The number of participants included in this analysis was 40.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.167 [60] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
19.81
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-8.7 | |||||||||||||||||||||||||||||||||
upper limit |
48.3 | |||||||||||||||||||||||||||||||||
Notes [60] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in adolescents at week 4. The number of participants included in this analysis was 20.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.005 [61] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
75.25
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
25.8 | |||||||||||||||||||||||||||||||||
upper limit |
124.7 | |||||||||||||||||||||||||||||||||
Notes [61] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in adolescents at week 8. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.033 [62] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
44.43
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
3.9 | |||||||||||||||||||||||||||||||||
upper limit |
84.9 | |||||||||||||||||||||||||||||||||
Notes [62] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in children at week 8. The number of participants included in this analysis was 41.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.02 [63] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
34.01
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
5.7 | |||||||||||||||||||||||||||||||||
upper limit |
62.3 | |||||||||||||||||||||||||||||||||
Notes [63] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in children at week 12. The number of participants included in this analysis was 39.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.503 [64] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
8.68
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-17.3 | |||||||||||||||||||||||||||||||||
upper limit |
34.7 | |||||||||||||||||||||||||||||||||
Notes [64] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in adolescents at week 12. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.016 [65] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
38.23
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
7.8 | |||||||||||||||||||||||||||||||||
upper limit |
68.6 | |||||||||||||||||||||||||||||||||
Notes [65] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in children at week 24. The number of participants included in this analysis was 36.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.043 [66] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
40.76
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
1.4 | |||||||||||||||||||||||||||||||||
upper limit |
80.2 | |||||||||||||||||||||||||||||||||
Notes [66] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in adolescents at week 24. The number of participants included in this analysis was 20.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [67] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
86.66
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
41.5 | |||||||||||||||||||||||||||||||||
upper limit |
131.8 | |||||||||||||||||||||||||||||||||
Notes [67] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in children at week 36. The number of participants included in this analysis was 37.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.203 [68] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
31.08
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-17.5 | |||||||||||||||||||||||||||||||||
upper limit |
79.6 | |||||||||||||||||||||||||||||||||
Notes [68] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in adolescents at week 36. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.019 [69] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
68.47
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
12.7 | |||||||||||||||||||||||||||||||||
upper limit |
124.2 | |||||||||||||||||||||||||||||||||
Notes [69] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in children at week 52. The number of participants included in this analysis was 39.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.042 [70] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
31.83
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
1.3 | |||||||||||||||||||||||||||||||||
upper limit |
62.4 | |||||||||||||||||||||||||||||||||
Notes [70] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in average morning catheterized volume in adolescents at week 52. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.121 [71] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
38.14
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-11 | |||||||||||||||||||||||||||||||||
upper limit |
87.3 | |||||||||||||||||||||||||||||||||
Notes [71] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Mean Number of Leakage Episodes per Day | |||||||||||||||||||||||||||||||||
End point description |
For each participant, the mean number of leakage episodes per day (during day & night time) was calculated using all available/non-missing number of leakage episodes for the 2 measuring days in the weekend diary during day & night time. If the number of leakage episodes was recorded on 1 single day in the 7-day diary during day & night time, the mean number of leakage episodes per day during day & night time is equal to the total number of leakage episodes recorded that day during day & night time. If no leakage episodes were recorded on any day of the weekend diary during day & night time, the mean number of leakage episodes per day was zero. Participants who did not report any leakage episode during the visit were imputed with a '0' for that visit. A valid bladder diary day in the weekend diary was any e-diary day for which ≥1 catheterized volume >0 mL was recorded with complete date and time. Analysis population consisted of FAS population with available data.
|
|||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||
End point timeframe |
Baseline and weeks 2, 4, 8, 12, 24, 36, and 52
|
|||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 2. The number of participants included in this analysis was 38.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.818 [72] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
0.35
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-2.7 | |||||||||||||||||||||||||||||||||
upper limit |
3.4 | |||||||||||||||||||||||||||||||||
Notes [72] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 2. The number of participants included in this analysis was 15.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.114 [73] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-0.53
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-1.2 | |||||||||||||||||||||||||||||||||
upper limit |
0.1 | |||||||||||||||||||||||||||||||||
Notes [73] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 4. The number of participants included in this analysis was 35.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.052 [74] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-1.14
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-2.3 | |||||||||||||||||||||||||||||||||
upper limit |
0 | |||||||||||||||||||||||||||||||||
Notes [74] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 4. The number of participants included in this analysis was 13.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.066 [75] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-0.87
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-1.8 | |||||||||||||||||||||||||||||||||
upper limit |
0.1 | |||||||||||||||||||||||||||||||||
Notes [75] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 8. The number of participants included in this analysis was 34.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.674 [76] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.16
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-4.4 | |||||||||||||||||||||||||||||||||
upper limit |
6.7 | |||||||||||||||||||||||||||||||||
Notes [76] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 8. The number of participants included in this analysis was 9.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.278 [77] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-0.65
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-1.9 | |||||||||||||||||||||||||||||||||
upper limit |
0.6 | |||||||||||||||||||||||||||||||||
Notes [77] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 12. The number of participants included in this analysis was 32.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.871 [78] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
0.37
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-4.2 | |||||||||||||||||||||||||||||||||
upper limit |
5 | |||||||||||||||||||||||||||||||||
Notes [78] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 12. The number of participants included in this analysis was 12.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.153 [79] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-0.65
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-1.6 | |||||||||||||||||||||||||||||||||
upper limit |
0.3 | |||||||||||||||||||||||||||||||||
Notes [79] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 24. The number of participants included in this analysis was 31.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.922 [80] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
0.18
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-3.5 | |||||||||||||||||||||||||||||||||
upper limit |
3.9 | |||||||||||||||||||||||||||||||||
Notes [80] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 24. The number of participants included in this analysis was 13.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.047 [81] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-0.75
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-1.5 | |||||||||||||||||||||||||||||||||
upper limit |
0 | |||||||||||||||||||||||||||||||||
Notes [81] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 36. The number of participants included in this analysis was 28.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.018 [82] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-1.98
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-3.6 | |||||||||||||||||||||||||||||||||
upper limit |
-0.4 | |||||||||||||||||||||||||||||||||
Notes [82] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 36. The number of participants included in this analysis was 12.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.07 [83] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-0.81
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-1.7 | |||||||||||||||||||||||||||||||||
upper limit |
0.1 | |||||||||||||||||||||||||||||||||
Notes [83] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 52. The number of participants included in this analysis was 27.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.083 [84] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-0.94
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-2 | |||||||||||||||||||||||||||||||||
upper limit |
0.1 | |||||||||||||||||||||||||||||||||
Notes [84] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 52. The number of participants included in this analysis was 13.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.064 [85] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
-1.12
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
-2.3 | |||||||||||||||||||||||||||||||||
upper limit |
0.1 | |||||||||||||||||||||||||||||||||
Notes [85] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Number of Dry Days per 7 Days (Day and Night Time) | |||||||||||||||||||||||||||||||||
End point description |
Dry days were defined as leakage-free days, this included day and night time. Participants recorded dry days in the 7-day diary. Dry days were calculated as follows: Ddry was the number of valid diary days where the response to the question ‘Did you leak between this catheterization and the last one’ was ‘No’ each time a new catheterization was entered in the e-diary during the day & night time period. Dwet was the number of valid diary days where the response to the question ‘Did you leak between this catheterization and the last one’ was ‘Yes’ for at least one catheterization entered during the day & night time period. If (Ddry + Dwet) > 3, the number of dry days per 7 days was calculated as Ddry/(Ddry + Dwet) x 7, otherwise the value was missing. Analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
|||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||
End point timeframe |
Baseline and weeks 2, 4, 8, 12, 24, 36, and 52
|
|||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in children at week 2. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.018 [86] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
0.34
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.1 | |||||||||||||||||||||||||||||||||
upper limit |
0.6 | |||||||||||||||||||||||||||||||||
Notes [86] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in adolescents at week 2. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.045 [87] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
0.82
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0 | |||||||||||||||||||||||||||||||||
upper limit |
1.6 | |||||||||||||||||||||||||||||||||
Notes [87] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in children at week 4. The number of participants included in this analysis was 42.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.013 [88] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
0.68
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.1 | |||||||||||||||||||||||||||||||||
upper limit |
1.2 | |||||||||||||||||||||||||||||||||
Notes [88] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in adolescents at week 4. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.002 [89] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.36
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.6 | |||||||||||||||||||||||||||||||||
upper limit |
2.2 | |||||||||||||||||||||||||||||||||
Notes [89] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in children at week 8. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.001 [90] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.14
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.5 | |||||||||||||||||||||||||||||||||
upper limit |
1.8 | |||||||||||||||||||||||||||||||||
Notes [90] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in adolescents at week 8. The number of participants included in this analysis was 23.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [91] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
2.26
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
1.2 | |||||||||||||||||||||||||||||||||
upper limit |
3.3 | |||||||||||||||||||||||||||||||||
Notes [91] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in children at week 12. The number of participants included in this analysis was 43.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.001 [92] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.31
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.5 | |||||||||||||||||||||||||||||||||
upper limit |
2.1 | |||||||||||||||||||||||||||||||||
Notes [92] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in children at week 24. The number of participants included in this analysis was 41.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [93] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.34
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.7 | |||||||||||||||||||||||||||||||||
upper limit |
2 | |||||||||||||||||||||||||||||||||
Notes [93] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in adolescents at week 12. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [94] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.93
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.9 | |||||||||||||||||||||||||||||||||
upper limit |
3 | |||||||||||||||||||||||||||||||||
Notes [94] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in adolescents at week 24. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [95] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
2.17
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
1.2 | |||||||||||||||||||||||||||||||||
upper limit |
3.2 | |||||||||||||||||||||||||||||||||
Notes [95] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in children at week 36. The number of participants included in this analysis was 39.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.002 [96] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.33
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.5 | |||||||||||||||||||||||||||||||||
upper limit |
2.1 | |||||||||||||||||||||||||||||||||
Notes [96] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in children at week 52. The number of participants included in this analysis was 40.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.002 [97] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.38
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
0.5 | |||||||||||||||||||||||||||||||||
upper limit |
2.2 | |||||||||||||||||||||||||||||||||
Notes [97] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in adolescents at week 36. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [98] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
1.88
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
1 | |||||||||||||||||||||||||||||||||
upper limit |
2.8 | |||||||||||||||||||||||||||||||||
Notes [98] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in number of dry days per 7 days (day and night time) in adolescents at week 52. The number of participants included in this analysis was 24.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
68
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [99] | |||||||||||||||||||||||||||||||||
Method |
Paired t-test | |||||||||||||||||||||||||||||||||
Parameter type |
Mean difference (net) | |||||||||||||||||||||||||||||||||
Point estimate |
2.14
|
|||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
level |
95% | |||||||||||||||||||||||||||||||||
sides |
2-sided
|
|||||||||||||||||||||||||||||||||
lower limit |
1.1 | |||||||||||||||||||||||||||||||||
upper limit |
3.2 | |||||||||||||||||||||||||||||||||
Notes [99] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
|||||||||||||||||||
End point title |
Change From Baseline in Pediatric Incontinence Questionnaire (PIN-Q) Score | ||||||||||||||||||
End point description |
PIN-Q measured quality of life via an e-diary. Total score ranged from 0/no effect to 80/worst effect; decrease in score indicated improvement. Total score was 20x average of individual PinQ items, the 20 Likert scales were converted to a score: Items 6 & 17; 0: “No” to 4: “Definitely” was used; & For the other 18 items; 0: “No” to 4: “All the time” was used. Expectation that questionnaires had limited missing values; if answers >2 questions were missing, total score was not calculated & was missing. Individual item scores were directly imputed. Change from baseline to each post-baseline visit in the total score was post-baseline visit value minus baseline value. If either baseline or post-baseline visit value was missing, change from baseline was missing. If change was: <0, improvement between 2 time-points; =0, no change between 2 time points; >0, worsening between 2 time points. FAS population. N is the number of participants with available data at each time point.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
Baseline and weeks 24 and 52
|
||||||||||||||||||
|
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in PIN-Q score in children at week 24. The number of participants included in this analysis was 24.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.352 [100] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
2.04
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-2.4 | ||||||||||||||||||
upper limit |
6.49 | ||||||||||||||||||
Notes [100] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in PIN-Q score in adolescents at week 24. The number of participants included in this analysis was 21.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.127 [101] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-4.9
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-11.34 | ||||||||||||||||||
upper limit |
1.53 | ||||||||||||||||||
Notes [101] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in PIN-Q score in children at week 52. The number of participants included in this analysis was 23.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.613 [102] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
1.3
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-3.96 | ||||||||||||||||||
upper limit |
6.57 | ||||||||||||||||||
Notes [102] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in PIN-Q score in adolescents at week 52. The number of participants included in this analysis was 19.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.056 [103] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
-6.79
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-13.78 | ||||||||||||||||||
upper limit |
0.2 | ||||||||||||||||||
Notes [103] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
|||||||||||||||||||
End point title |
Change From Baseline in Patient Global Impression of Severity Scale (PGI-S) | ||||||||||||||||||
End point description |
The PGI-S is an answer to the question: “How did you feel about your bladder condition during the past 3 days?” Participants evaluated their recent condition as “Really Bad”(0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). An increase indicated improvement. The change from baseline to each postbaseline visit in the PGI-S score is the value at the post-baseline visit minus the value at the baseline visit. If either the baseline or the post-baseline visit value is missing, the change from baseline was missing. A positive change indicated an improvement while a negative change indicated a worsening. Analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
Baseline and weeks 24 and 52
|
||||||||||||||||||
|
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in PGI-S in children at week 24. The number of participants included in this analysis was 25.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.153 [104] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
0.36
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-0.14 | ||||||||||||||||||
upper limit |
0.86 | ||||||||||||||||||
Notes [104] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in PGI-S in adolescents at week 24. The number of participants included in this analysis was 22.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.007 [105] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
0.64
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
0.19 | ||||||||||||||||||
upper limit |
1.08 | ||||||||||||||||||
Notes [105] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in PGI-S in children at week 52. The number of participants included in this analysis was 24.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.106 [106] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
0.42
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
-0.1 | ||||||||||||||||||
upper limit |
0.93 | ||||||||||||||||||
Notes [106] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in PGI-S in adolescents at week 52. The number of participants included in this analysis was 19.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.003 [107] | ||||||||||||||||||
Method |
Paired t-test | ||||||||||||||||||
Parameter type |
Mean difference (net) | ||||||||||||||||||
Point estimate |
0.95
|
||||||||||||||||||
Confidence interval |
|||||||||||||||||||
level |
95% | ||||||||||||||||||
sides |
2-sided
|
||||||||||||||||||
lower limit |
0.38 | ||||||||||||||||||
upper limit |
1.51 | ||||||||||||||||||
Notes [107] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Clinician Global Impression of Change (CGI-C) | |||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
The Clinician Global Impression of Change (CGI-C) is a 7 point scale that requires the clinician to assess how much the participant’s overall bladder symptoms since the start of the study on day 1 has improved or worsened and rated as: very much improved (1); much improved (2); minimally improved (3); no change (4); minimally worse (5); much worse (6); or very much worse (7). Analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Weeks 24 and 52
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||||||||||||||||||||||||||
End point title |
Study Drug Acceptability for Tablets at Week 4 | |||||||||||||||||||||||||||||||||||||||
End point description |
Participants evaluated the taste of the study medication/tablets by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the swallow of the study medication/tablets by ticking one of the following categories: “Really Difficult” (0), “Difficult” (1), “Not Difficult, Not Easy” (2), “Easy” (3) and “Really Easy” (4). Analysis population consisted of the FAS (participants on tablets with available data at week 4).
|
|||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||
End point timeframe |
Week 4
|
|||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Study Drug Acceptability for Oral Suspension at Week 4 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Participants evaluated the taste of the study medication/oral suspension by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the smell of the study medication/oral suspension by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the consumption and the preparation of the study medication/oral suspension by ticking 1 of the following categories: “Really Difficult” (0), “Difficult” (1), “Not Difficult, Not Easy” (2), “Easy” (3) & “Really Easy” (4). Analysis population consisted of the FAS (participants on oral suspension with available data at week 4).
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Week 4
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||||||||||||||||||||||||||
End point title |
Study Drug Acceptability for Tablets at Week 24 | |||||||||||||||||||||||||||||||||||||||
End point description |
Participants evaluated the taste of the study medication/tablets by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the swallow of the study medication/tablets by ticking one of the following categories: “Really Difficult” (0), “Difficult” (1), “Not Difficult, Not Easy” (2), “Easy” (3) and “Really Easy” (4). Analysis population consisted of the FAS (participants on tablets with available data at week 24).
|
|||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||
End point timeframe |
Week 24
|
|||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Study Drug Acceptability for Oral Suspension at Week 24 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Participants evaluated the taste of the study medication/oral suspension by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the smell of the study medication/oral suspension by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the consumption and the preparation of the study medication/oral suspension by ticking 1 of the following categories: “Really Difficult” (0), “Difficult” (1), “Not Difficult, Not Easy” (2), “Easy” (3) & “Really Easy” (4). Analysis population consisted of the FAS (participants on oral suspension with available data at week 24).
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Week 24
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||||||||||||||||||||||||||
End point title |
Study Drug Acceptability for Tablets at Week 52 | |||||||||||||||||||||||||||||||||||||||
End point description |
Participants evaluated the taste of the study medication/tablets by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the swallow of the study medication/tablets by ticking one of the following categories: “Really Difficult” (0), “Difficult” (1), “Not Difficult, Not Easy” (2), “Easy” (3) and “Really Easy” (4). Analysis population consisted of the FAS (participants on tablets with available data at week 52).
|
|||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||
End point timeframe |
Week 52
|
|||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Study Drug Acceptability for Oral Suspension at Week 52 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Participants evaluated the taste of the study medication/oral suspension by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the smell of the study medication/oral suspension by ticking 1 of the following categories: “Really Bad” (0), “Bad” (1), “Not Bad, Not Good” (2), “Good” (3) & “Really Good” (4). Participants evaluated the consumption and the preparation of the study medication/oral suspension by ticking 1 of the following categories: “Really Difficult” (0), “Difficult” (1), “Not Difficult, Not Easy” (2), “Easy” (3) & “Really Easy” (4). Analysis population consisted of the FAS (participants on oral suspension with available data at week 52).
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Week 52
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||||||||||||||||||||||||||
End point title |
Number of Participants with Adverse Events (AEs) | ||||||||||||||||||||||||||||||||||||
End point description |
An AE was defined as any untoward medical occurrence in a participant who was given the study drug or who had undergone study procedures and did not necessarily have a causal relationship with this treatment. An AE could therefore be any unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A treatment-emergent adverse event (TEAE) was defined as any AE with date of onset occurring on or after the first dose of study medication and up to the end of study. The analysis population consisted of the safety analysis set (SAF), which consisted of all participants who took at least 1 dose of study drug.
|
||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||||||||
End point timeframe |
From the first dose of study drug administration up to end-of-treatment (EoT) (week 52).
|
||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||||||||||
End point title |
Pharmacokinetics (PK) of Maximum Plasma Mirabegron Concentration (Cmax) | ||||||||||||||||||||
End point description |
The analysis population consisted of the pharmacokinetic analysis set (PKAS), which consisted of the subset of the SAF for whom plasma concentration data were available to facilitate derivation of ≥ 1 pharmacokinetic parameter and for whom the time of the last dose prior to sampling was known. The population of the pharmacokinetic analysis included all participants (n=71) that contributed at least one measurable PK sample. This included 5 partcipants who were excluded from the PKAS dataset (n=66) because PK samples were collected outside the protocol specified time window for collection. Due to the low number of participants, data could not be calculated and is denoted as "99999" as applicable.
|
||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||
End point timeframe |
A total of 4 samples were collected over 2 sampling days at 2 separate visits at any of weeks 4, 8, 12, 24, 36, or 52, at the following time points: Sampling day 1, predose; Sampling day 2, predose and 2 samples 2-5 hours postdose more than 1 hour apart.
|
||||||||||||||||||||
|
|||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||||||||||
End point title |
Time to Reach Maximum Plasma Concentration of Mirabegron Following Drug Administration (Tmax) | ||||||||||||||||||||
End point description |
The analysis population consisted of the PKAS. The population of the pharmacokinetic analysis included all participants (n=71) that contributed at least one measurable PK sample. This included 5 partcipants who were excluded from the PKAS dataset (n=66) because PK samples were collected outside the protocol specified time window for collection. Due to the low number of participants, data could not be calculated and is denoted as "99999" as applicable.
|
||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||
End point timeframe |
A total of 4 samples were collected over 2 sampling days at 2 separate visits at any of weeks 4, 8, 12, 24, 36, or 52, at the following time points: Sampling day 1, predose; Sampling day 2, predose and 2 samples 2-5 hours postdose more than 1 hour apart.
|
||||||||||||||||||||
|
|||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||||||||||
End point title |
Area Under the Plasma Concentration-Time Curve From Time Zero to 24 Hours (AUC24) for Mirabegron | ||||||||||||||||||||
End point description |
The analysis population consisted of the PKAS. The population of the pharmacokinetic analysis included all participants (n=71) that contributed at least one measurable PK sample. This included 5 partcipants who were excluded from the PKAS dataset (n=66) because PK samples were collected outside the protocol specified time window for collection. Due to the low number of participants, data could not be calculated and is denoted as "99999" as applicable.
|
||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||
End point timeframe |
A total of 4 samples were collected over 2 sampling days at 2 separate visits at any of weeks 4, 8, 12, 24, 36, or 52, at the following time points: Sampling day 1, predose; Sampling day 2, predose and 2 samples 2-5 hours postdose more than 1 hour apart.
|
||||||||||||||||||||
|
|||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||||||||||
End point title |
Plasma Concentration of Mirabegron at the End of a Dosing interval at Steady State (Ctrough) | ||||||||||||||||||||
End point description |
The analysis population consisted of the PKAS. The population of the pharmacokinetic analysis included all participants (n=71) that contributed at least one measurable PK sample. This included 5 partcipants who were excluded from the PKAS dataset (n=66) because PK samples were collected outside the protocol specified time window for collection. Due to the low number of participants, data could not be calculated and is denoted as "99999" as applicable.
|
||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||
End point timeframe |
A total of 4 samples were collected over 2 sampling days at 2 separate visits at any of weeks 4, 8, 12, 24, 36, or 52, at the following time points: Sampling day 1, predose; Sampling day 2, predose and 2 samples 2-5 hours postdose more than 1 hour apart.
|
||||||||||||||||||||
|
|||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||||||||||
End point title |
Apparent Total Clearance of Mirabegron From Plasma After Oral Administration (CL/F) | ||||||||||||||||||||
End point description |
The analysis population consisted of the PKAS. The population of the pharmacokinetic analysis included all participants (n=71) that contributed at least one measurable PK sample. This included 5 partcipants who were excluded from the PKAS dataset (n=66) because PK samples were collected outside the protocol specified time window for collection. Due to the low number of participants, data could not be calculated and is denoted as "99999" as applicable.
|
||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||
End point timeframe |
A total of 4 samples were collected over 2 sampling days at 2 separate visits at any of weeks 4, 8, 12, 24, 36, or 52, at the following time points: Sampling day 1, predose; Sampling day 2, predose and 2 samples 2-5 hours postdose more than 1 hour apart.
|
||||||||||||||||||||
|
|||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||||||||||
End point title |
Apparent Volume of Distribution After Non-intravenous Administration (Vz/F) of Mirabegron | ||||||||||||||||||||
End point description |
The analysis population consisted of the PKAS. The population of the pharmacokinetic analysis included all participants (n=71) that contributed at least one measurable PK sample. This included 5 partcipants who were excluded from the PKAS dataset (n=66) because PK samples were collected outside the protocol specified time window for collection. Due to the low number of participants, data could not be calculated and is denoted as "99999" as applicable.
|
||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||
End point timeframe |
A total of 4 samples were collected over 2 sampling days at 2 separate visits at any of weeks 4, 8, 12, 24, 36, or 52, at the following time points: Sampling day 1, predose; Sampling day 2, predose and 2 samples 2-5 hours postdose more than 1 hour apart.
|
||||||||||||||||||||
|
|||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||||||||
End point title |
Change From Baseline in Filling Bladder Volume Until First Overactive Detrusor Contraction (> 15 cm H20) | ||||||||||||||||||
End point description |
Detrusor overactivity is the occurrence of involuntary detrusor contractions during filling cystometry. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. The analysis population consisted of the FAS. N is the number of participants with available data at each time point. If no detrusor contraction of > 15 cm H2O occurred, the bladder volume was imputed with maximum cystometric capacity.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
Baseline and weeks 4 and 24
|
||||||||||||||||||
|
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in children at week 4. The number of participants included in this analysis was 21.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [108] | ||||||||||||||||||
Method |
Wilcoxon signed-rank test | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [108] - From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in adolescents at week 4. The number of participants included in this analysis was 8.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.148 [109] | ||||||||||||||||||
Method |
Wilcoxon signed-rank test | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [109] - From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in children at week 24. The number of participants included in this analysis was 13.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.002 [110] | ||||||||||||||||||
Method |
Wilcoxon signed-rank test | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [110] - From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in adolescents at week 24. The number of participants included in this analysis was 8.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
68
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.039 [111] | ||||||||||||||||||
Method |
Wilcoxon signed-rank test | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [111] - From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median. |
|
|||||||||||||||||||
End point title |
Change From Baseline in Filling Bladder Volume Until First Overactive Detrusor Contraction (> 15 cm H20): Wilcoxon signed-rank Test Updated Analysis | ||||||||||||||||||
End point description |
Detrusor overactivity is occurrence of involuntary detrusor contractions during filling cystometry. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. If no detrusor contraction of > 15 cm H2O occurred, the bladder volume was imputed with maximum cystometric capacity. This updated analysis is presented as the original analysis of bladder volume until first detrusor contraction (> 15 cm H2O) did not impute missing bladder volume data with the MCC value at that visit according to the SAP. This analysis was updated to impute missing values for volume at first contraction with respective MCC values. The analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
Baseline and weeks 4 and 24
|
||||||||||||||||||
|
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in children at week 4. The number of participants included in this analysis was 41.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [112] | ||||||||||||||||||
Method |
Wilcoxon signed-rank test | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [112] - From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in adolescents at week 24. The number of participants included in this analysis was 24.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [113] | ||||||||||||||||||
Method |
Wilcoxon signed-rank test | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [113] - From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in children at week 24. The number of participants included in this analysis was 38.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [114] | ||||||||||||||||||
Method |
Wilcoxon signed-rank test | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [114] - From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in adolescents at week 4. The number of participants included in this analysis was 23.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.007 [115] | ||||||||||||||||||
Method |
Wilcoxon signed-rank test | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [115] - From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median. |
|
|||||||||||||||||||
End point title |
Change From Baseline in Filling Bladder Volume Until First Overactive Detrusor Contraction (> 15 cm H20): Paired t-test | ||||||||||||||||||
End point description |
Detrusor overactivity is the occurrence of involuntary detrusor contractions during filling cystometry. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. If no detrusor contraction of > 15 cm H2O occurred, the bladder volume was imputed with maximum cystometric capacity. The analysis population consisted of the FAS. N is the number of participants with available data at each time point.
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
Baseline and weeks 4 and 24
|
||||||||||||||||||
|
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in children at week 4. The number of participants included in this analysis was 41.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [116] | ||||||||||||||||||
Method |
t-test, 2-sided | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [116] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline was equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in children at week 24. The number of participants included in this analysis was 38.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
< 0.001 [117] | ||||||||||||||||||
Method |
t-test, 2-sided | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [117] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline was equal to 0. |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in adolescents at week 24. The number of participants included in this analysis was 24.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.001 | ||||||||||||||||||
Method |
t-test, 2-sided | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | ||||||||||||||||||
Statistical analysis description |
Change from baseline in filling bladder volume until first overactive detrusor contraction in adolescents at week 4. The number of participants included in this analysis was 23.
|
||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
||||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||||
Analysis type |
other | ||||||||||||||||||
P-value |
= 0.006 [118] | ||||||||||||||||||
Method |
t-test, 2-sided | ||||||||||||||||||
Confidence interval |
|||||||||||||||||||
Notes [118] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline was equal to 0. |
|
||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Mean Number of Leakage Episodes per Day: Updated Analysis | |||||||||||||||||||||||||||||||||
End point description |
The mean number(no.) of leakage episodes per day (during day & night time (D&N)) was calculated using all available/non-missing no. of leakage episodes for the 2 measuring days in the weekend diary during day & night time. If the no. of leakage episodes was recorded on 1 single day in the 7-day diary D&N, the mean no. of leakage episodes per day D&N is equal to the total no. of leakage episodes recorded that day D&N. If no leakage episodes were recorded on any day of the weekend diary, the mean no. of leakage episodes per day was zero. Participants who did not report leakage episode during the visit were imputed with a '0' for that visit. Valid bladder diary day in weekend diary was any e-diary day for which ≥1 catheterized volume >0 mL was recorded with complete date and time. Updated analysis is presented because one participant entered weight of leakage instead of no. of leakages. FAS Population. N is the number of participants with available data at each time point.
|
|||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||
End point timeframe |
Baseline and weeks 2, 4, 8, 12, 24, 36 and 52
|
|||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 2. The number of participants included in this analysis was 30.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.692 [119] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [119] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 2 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 2. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.018 [120] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [120] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 4. The number of participants included in this analysis was 29.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.061 [121] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [121] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 4 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 4. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.008 [122] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [122] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0 |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 8. The number of participants included in this analysis was 29.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.612 [123] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [123] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 8 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 8. The number of participants included in this analysis was 19.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.018 [124] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [124] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 12. The number of participants included in this analysis was 28.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.858 | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 24. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
< 0.001 [125] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [125] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 24 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 24. The number of participants included in this analysis was 27.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.003 [126] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [126] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 12 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 12. The number of participants included in this analysis was 21.
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.004 [127] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [127] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 52. The number of participants included in this analysis was 27
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.006 [128] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [128] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 36. The number of participants included in this analysis was 21
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.002 [129] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [129] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 36 - Children | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in children at week 36. The number of participants included in this analysis was 26
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.045 [130] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [130] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
||||||||||||||||||||||||||||||||||
Statistical analysis title |
Change From Baseline at Week 52 - Adolescents | |||||||||||||||||||||||||||||||||
Statistical analysis description |
Change from baseline in mean number of leakage episodes per day in adolescents at week 52. The number of participants included in this analysis was 21
|
|||||||||||||||||||||||||||||||||
Comparison groups |
Children (3 to < 12 Years) v Adolescents (12 to < 18 Years)
|
|||||||||||||||||||||||||||||||||
Number of subjects included in analysis |
51
|
|||||||||||||||||||||||||||||||||
Analysis specification |
Pre-specified
|
|||||||||||||||||||||||||||||||||
Analysis type |
other | |||||||||||||||||||||||||||||||||
P-value |
= 0.007 [131] | |||||||||||||||||||||||||||||||||
Method |
t-test, 2-sided | |||||||||||||||||||||||||||||||||
Confidence interval |
||||||||||||||||||||||||||||||||||
Notes [131] - From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0. |
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Adverse events information
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
From the first dose of study drug administration up to end-of-treatment (EoT) (week 52).
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
16
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Adolescents (12 to < 18 Years)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Adolescents age 12 to < 18 received initial dose of mirabegron based on weight (pediatric equivalent dose [PED25]) on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults (PED50) based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Children (3 to < 12 Years)
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Reporting group description |
Children age 3 to < 12 received initial dose of mirabegron based on weight (pediatric equivalent dose [PED25]) on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults (PED50) based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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02 Nov 2016 |
The changes included: ● The age range of the patients was updated to include children from 3 to < 18 years of age. This study is targeted to fulfill both the EMA and FDA requirements related to the conduct of pediatric studies. A Written Request received from the FDA included a stipulation to decrease the lower limit of the age range from 5 to 3 years of age. ● Mirabegron oral suspension (8 mg/mL) was added to the protocol as a second study drug formulation to enable dosing of the younger patients. ● The weight range for inclusion of patients in the study was lowered to ≥ 11 kg. ● Additional SBPM were included at weeks 1 and 2 after the start of dosing and after a dose escalation to satisfy EMA and FDA comments. ● Nonsubstantial changes corrected administrative-type information. These protocol changes were implemented after enrollment of 38 patients |
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Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |