Clinical Trial Results:
A Multicentre, Long-term Safety, Efficacy and Pharmacokinetics Study of Lubiprostone in Paediatric Subjects Aged ≥6 to <18 years with Functional Constipation
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Summary
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EudraCT number |
2013-004384-31 |
Trial protocol |
BE GB DE ES NL FR PL |
Global end of trial date |
01 May 2017
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Results information
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Results version number |
v1(current) |
This version publication date |
19 Aug 2017
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First version publication date |
19 Aug 2017
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Other versions |
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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 |
SAG/0211PFC-11S1
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
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WHO universal trial number (UTN) |
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Sponsors
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Sponsor organisation name |
Sucampo Pharma Americas LLC
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Sponsor organisation address |
850 King Farm Blvd, Suite 550, Rockville, United States, MD, 20850
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Public contact |
Senior Director Regulatory Affairs, Sucampo AG, 41 417263045, hschulze@sucampo.com
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Scientific contact |
Senior Director Regulatory Affairs, Sucampo AG, 41 417263045, hschulze@sucampo.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-000245-PIP01-08 | ||
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 |
01 May 2017
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
01 May 2017
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Global end of trial reached? |
Yes
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Global end of trial date |
01 May 2017
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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 |
To assess the long-term safety, efficacy, and pharmacokinetics of oral lubiprostone 12 or 24 mcg capsules dosed twice daily (BID) when administered orally for 36 weeks in paediatric subjects with functional constipation.
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Protection of trial subjects |
none specific
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Background therapy |
none | ||
Evidence for comparator |
none. This was a single arm study with lubiprostone as the investigational medicinal product. | ||
Actual start date of recruitment |
26 Feb 2014
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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 |
Netherlands: 36
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Country: Number of subjects enrolled |
Poland: 10
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Country: Number of subjects enrolled |
United Kingdom: 10
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Country: Number of subjects enrolled |
Belgium: 2
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Country: Number of subjects enrolled |
France: 1
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Country: Number of subjects enrolled |
United States: 359
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Worldwide total number of subjects |
418
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EEA total number of subjects |
59
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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) |
232
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Adolescents (12-17 years) |
186
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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 |
Subjects who completed the placebo-controlled study SAG/0211PFC-1131 (3 months) were invited to enrol in this open-label long-term safety extension study (9 additional months). Also subjects in the placebo arm were able to participate in this study. The first patient was randomised on 27 March 2013. | ||||||||||||||||||||||||
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Pre-assignment
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Screening details |
Subjects must have completed the entire 12-week treatment period during the preceding study SAG/0211PFC-1131, agreed to continue to abstain from concomitant medication (prescribed or OTC) use that could affect gastrointestinal motility, were willing and able to administer rescue medication, if needed, and subject (or parent) must have given consent | ||||||||||||||||||||||||
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Period 1
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Period 1 title |
Overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||||||||||||
Blinding implementation details |
the study was not blinded.
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Arms
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Arm title
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study arm | ||||||||||||||||||||||||
Arm description |
This was a single arm study. All patients received lubiprostone. Those who received 12 mcg BID in the preceding study SAG/0211PFC-1131 continued to receive 12 mcg, those who received 24 mcg BID continued with 24 mcg. Those of the placebo arm weighing less than 50 kg received 12 mcg BID and those over 50 kg received 24 mcg BID. | ||||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||||
Investigational medicinal product name |
Lubiprostone
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Capsule, soft
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Routes of administration |
Oral use
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Dosage and administration details |
12 or 24 mcg BID. Those patients who received 12 mcg BID in the preceding study SAG/0211PFC-1131 continued to receive 12 mcg BID and those who received 24 mcg BID in the preceding study continued with 24 mcg BID. Patients from the placebo arm of the preceding study received 12 mcg BID when they weighed less than 50 kg, and 24 mcg BID when their weigt was greater than 50 kg.
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Baseline characteristics reporting groups
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Reporting group title |
Overall trial
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Reporting group description |
- | ||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
mITT Population
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Subject analysis set type |
Modified intention-to-treat | ||||||||||||||||||||||||||||||
Subject analysis set description |
the mITT group comprised all subjects who enroled in the trial, took at least one dose of study medication and had at least one diary entry after treatment initiation.
There was only one subject that did not fulfill these conditions, because of serious compliance issues with potential data integrity impact at the site.
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End points reporting groups
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Reporting group title |
study arm
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Reporting group description |
This was a single arm study. All patients received lubiprostone. Those who received 12 mcg BID in the preceding study SAG/0211PFC-1131 continued to receive 12 mcg, those who received 24 mcg BID continued with 24 mcg. Those of the placebo arm weighing less than 50 kg received 12 mcg BID and those over 50 kg received 24 mcg BID. | ||
Subject analysis set title |
mITT Population
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Subject analysis set type |
Modified intention-to-treat | ||
Subject analysis set description |
the mITT group comprised all subjects who enroled in the trial, took at least one dose of study medication and had at least one diary entry after treatment initiation.
There was only one subject that did not fulfill these conditions, because of serious compliance issues with potential data integrity impact at the site.
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End point title |
Change in number of spontaneous bowel movements (SBM) [1] | ||||||||||||||||
End point description |
Monthly and overall change from baseline in Spontaneous Bowel Movement (SBM) frequency.
Here the mean change of overall number of of SBM per week compared to baseline is provided.
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End point type |
Primary
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End point timeframe |
At Months 1 to 9
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Since this was an open-label study, descriptive statistics was used. |
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End point title |
Straining | ||||||||||||
End point description |
Monthly and overall change from baseline in straining associated with SBMs.
Here the change from baseline in straining is provided with a mean value of a 5-digit score.
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End point type |
Other pre-specified
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End point timeframe |
Months 1 to 9
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Stool consistency | ||||||||||||
End point description |
Monthly and overlall change from baseline in stool consistency associated with SBMs.
Here the overall change from baseline in stool consistency id provided as mean value of a 5-point rating scale.
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End point type |
Other pre-specified
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End point timeframe |
Months 1 to 9
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Abdominal pain | ||||||||||||
End point description |
Monthly and overall change from baseline in abdominal pain.
Here the overall change from baseline in abdominal pain is expressed as mean value of a 5-point scale.
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End point type |
Other pre-specified
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End point timeframe |
Months 1 to 9
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| No statistical analyses for this end point | |||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
9 conscutive months of study treatment followed by 1 month of follow-up
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Adverse event reporting additional description |
Patients or caregivers could report AEs at any time. Patients and caregivers were routinely asked about any AEs at each visit.
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Assessment type |
Non-systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
14
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Reporting groups
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Reporting group title |
Treatment group
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Reporting group description |
There was only one arm in this open-label study. All subjects received lubiprostone. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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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26 Nov 2013 |
clarification in exclusion criteria regarding pregnancy test, and measures to reduce stress and pain in paeditaric population |
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05 Dec 2013 |
Clarifications on study objectives and endpoints |
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19 Nov 2015 |
Increase number of sites. Some administrative changes and clarifications on inclusion criteria. |
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20 Sep 2016 |
change of sponsor, addition of a legal representative |
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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. | |||
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