Clinical Trial Results:
A 24-week Safety, Efficacy, Pharmacodynamic, and Pharmacokinetic Study of Teduglutide in
Japanese Pediatric Subjects, Aged 4 Months through 15 Years, with Short Bowel Syndrome who are Dependent on Parenteral Support
Summary
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EudraCT number |
2020-005791-35 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
21 Jan 2020
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Results information
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Results version number |
v1(current) |
This version publication date |
04 Feb 2021
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First version publication date |
04 Feb 2021
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Other versions |
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 |
SHP633-302
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02980666 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Shire
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Sponsor organisation address |
300 Shire Way, Lexington, United States, MA 02421
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Public contact |
Study Director, Shire, +1 866 842 5335, ClinicalTransparency@shire.com
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Scientific contact |
Study Director, Shire, +1 866 842 5335, ClinicalTransparency@shire.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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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? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
21 Jan 2020
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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 |
21 Jan 2020
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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 safety, tolerability, efficacy, pharmacodynamics (PD), and pharmacokinetics (PK) of teduglutide in Japanese pediatric subjects (4 months through 15 years of age) with short bowel syndrome (SBS) who were dependent on parenteral support (PS).
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Protection of trial subjects |
The study was conducted in accordance with current applicable industry regulations, International Council for Harmonisation (ICH) Good Clinical Practice (GCP) Guideline E6 (1996) and any updates, European Union (EU) Directive 2001/20/EC and its updates, the ethical principles in the Declaration of Helsinki, and local ethical and legal requirements.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
13 Jan 2017
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Long term follow-up planned |
Yes
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Long term follow-up rationale |
Efficacy, Safety | ||
Long term follow-up duration |
9 Months | ||
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 |
Japan: 10
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Worldwide total number of subjects |
10
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EEA total number of subjects |
0
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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) |
2
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Children (2-11 years) |
8
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Adolescents (12-17 years) |
0
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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 |
The study was conducted at 6 centers in Japan between 13 January 2017 (first subject first visit) and 21 January 2020 (last subject last visit). | |||||||||
Pre-assignment
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Screening details |
A total of 10 Japanese subjects were enrolled into the study, including 8 children age 1 through 15 years of age and 2 infants aged 4 months through < 12 months of corrected gestational age. All the subjects received treatment and completed the study. | |||||||||
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 |
Not applicable
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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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Total Children (Aged: 1 to 15 Years) | |||||||||
Arm description |
Subjects aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
Teduglutide
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Investigational medicinal product code |
SHP633
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Other name |
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Pharmaceutical forms |
Injection
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
Subjects received teduglutide 0.05 mg/kg/day SC injection into either thigh or arm or 1 of 4 quadrants of the abdomen.
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Arm title
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Infants (Corrected Gestational Age: 4 to < 12 months) | |||||||||
Arm description |
Subjects (Infants) from 4 through < 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
Teduglutide
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Investigational medicinal product code |
SHP633
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Other name |
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Pharmaceutical forms |
Injection
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
Subjects received teduglutide 0.05 mg/kg/day SC injection into either thigh or arm or 1 of 4 quadrants of the abdomen.
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Baseline characteristics reporting groups
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Reporting group title |
Total Children (Aged: 1 to 15 Years)
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Reporting group description |
Subjects aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Infants (Corrected Gestational Age: 4 to < 12 months)
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Reporting group description |
Subjects (Infants) from 4 through < 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Total Children (Aged: 1 to 15 Years)
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Reporting group description |
Subjects aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28. | ||
Reporting group title |
Infants (Corrected Gestational Age: 4 to < 12 months)
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Reporting group description |
Subjects (Infants) from 4 through < 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28. |
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End point title |
Absolute Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data [1] | ||||||||||||
End point description |
Absolute change from baseline in PS volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Here, milliliter per kilogram per day is abbreviated as mL/kg/day. Intention-to-treat (ITT) population consisted of all subjects who were enrolled into the study and met all eligible criteria for the study.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Percent Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data [2] | ||||||||||||
End point description |
Percent change from baseline in PS volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for the study.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [2] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Absolute Change From Baseline in Parenteral Support (PS) Caloric Intake at End of Treatment (EOT) Based on Dairy Data [3] | ||||||||||||
End point description |
Absolute change from baseline in PS caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day). ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for the study.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [3] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Percent Change From Baseline in Parenteral Support (PS) Caloric Intake at End of Treatment (EOT) Based on Dairy Data [4] | ||||||||||||
End point description |
Percent change from baseline in PS caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for the study.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [4] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Absolute Change From Baseline in Plasma Citrulline at End of Treatment (EOT) [5] | ||||||||||||
End point description |
Absolute change from baseline in plasma citrulline at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for the study.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [5] - 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: No statistical and comparison analyses were performed for this endpoint. |
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Notes [6] - Data for this end point was not planned to be collected and analysed for infants. |
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No statistical analyses for this end point |
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End point title |
Percent Change From Baseline in Plasma Citrulline at End of Treatment (EOT) [7] | ||||||||||||
End point description |
Percent change from baseline in plasma citrulline at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for the study.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [7] - 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: No statistical and comparison analyses were performed for this endpoint. |
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Notes [8] - Data for this endpoint was not planned to be collected and analysed for infants. |
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No statistical analyses for this end point |
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End point title |
Absolute Change From Baseline in Enteral Nutritional (EN) Volume at End of Treatment (EOT) Based on Dairy Data [9] | ||||||||||||
End point description |
Absolute change from baseline in EN volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [9] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Percent Change From Baseline in Enteral Nutritional (EN) Volume at End of Treatment (EOT) Based on Dairy Data [10] | ||||||||||||
End point description |
Percent change from baseline in EN volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [10] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Absolute Change From Baseline in Enteral Nutritional (EN) Caloric Intake at End of Treatment (EOT) Based on Dairy Data [11] | ||||||||||||
End point description |
Absolute change from baseline in EN caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [11] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Percent Change From Baseline in Enteral Nutritional (EN) Caloric Intake at End of Treatment (EOT) Based on Dairy Data [12] | ||||||||||||
End point description |
Percent change from baseline in EN caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to Week 24)
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Notes [12] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Number of Subjects Who Achieved At least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at Week 24 [13] | |||||||||
End point description |
Number of subjects who achieved at least 20% reduction in PS volume at Week 24 was reported. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
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End point type |
Primary
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End point timeframe |
Week 24
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Notes [13] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Number of Subjects Who Achieved At least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at End of Treatment (EOT) [14] | |||||||||
End point description |
Number of subjects who achieved at least 20% reduction in PS volume at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
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End point type |
Primary
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End point timeframe |
EOT (up to Week 24)
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Notes [14] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Number of Subjects Who Achieved 100 Percent (%) Reduction in Complete Weaning of Parenteral Support (PS) Volume at End of Treatment (EOT) [15] | |||||||||
End point description |
Number of subjects who achieved at least 100% reduction in complete weaning of PS volume at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
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End point type |
Primary
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End point timeframe |
EOT (up to Week 24)
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Notes [15] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Number of Subjects Who Achieved Greater Than or Equal to (>=) 20 Percent (%) Reduction in Parenteral Support (PS) Volume at Week 28 [16] | |||||||||
End point description |
Number of subjects who achieved >= 20% reduction in PS volume at Week 28 was reported. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
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End point type |
Primary
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End point timeframe |
Week 28
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Notes [16] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Absolute Change From End of Treatment (EOT) in Parenteral Support (PS) Volume at End of Study (EOS) Based on Dairy Data [17] | ||||||||||||
End point description |
Absolute change from EOT (up to Week 24) in PS volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
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End point type |
Primary
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End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
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Notes [17] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Percent Change From End of Treatment (EOT) in Parenteral Support (PS) Volume at End of Study (EOS) Based on Dairy Data [18] | ||||||||||||
End point description |
Percent change from EOT (up to Week 24) in PS volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
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End point type |
Primary
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End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
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Notes [18] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Absolute Change From End of Treatment (EOT) in Parenteral Support (PS) Caloric Intake at End of Study (EOS) Based on Dairy Data [19] | ||||||||||||
End point description |
Absolute change from EOT (up to Week 24) in PS caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
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End point type |
Primary
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End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
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Notes [19] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Percent Change From End of Treatment (EOT) in Parenteral Support (PS) Caloric Intake at End of Study (EOS) Based on Dairy Data [20] | ||||||||||||
End point description |
Percent change from EOT (up to Week 24) in PS caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
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End point type |
Primary
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End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
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Notes [20] - 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: No statistical and comparison analyses were performed for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Absolute Change From End of Treatment (EOT) in Plasma Citrulline at End of Study (EOS) [21] | ||||||||||||
End point description |
Absolute change from EOT (up to Week 24) in plasma citrulline at EOS (up to Week 28) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
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End point type |
Primary
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End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
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Notes [21] - 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: No statistical and comparison analyses were performed for this endpoint. |
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Notes [22] - Data for this endpoint was not planned to be collected and analysed for infants. |
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No statistical analyses for this end point |
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End point title |
Percent Change From End of Treatment (EOT) in Plasma Citrulline at End of Study (EOS) [23] | ||||||||||||
End point description |
Percent change from EOT (up to Week 24) in plasma citrulline at EOS (up to Week 28) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
|
||||||||||||
Notes [23] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [24] - Data for this endpoint was not planned to be collected and analysed for infants. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Absolute Change From End of Treatment (EOT) in Enteral Nutritional (EN) Volume at End of Study (EOS) Based on Dairy Data [25] | ||||||||||||
End point description |
Absolute change from EOT (up to Week 24) in EN volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
|
||||||||||||
Notes [25] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Percent Change From End of Treatment (EOT) in Enteral Nutritional (EN) Volume at End of Study (EOS) Based on Dairy Data [26] | ||||||||||||
End point description |
Percent change from EOT (up to Week 24) in EN volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
|
||||||||||||
Notes [26] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Absolute Change From End of Treatment (EOT) in Enteral Nutritional (EN) Caloric Intake at End of Study (EOS) Based on Dairy Data [27] | ||||||||||||
End point description |
Absolute change from EOT (up to Week 24) in EN caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
|
||||||||||||
Notes [27] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Percent Change From End of Treatment (EOT) in Enteral Nutritional (EN) Caloric Intake at End of Study (EOS) Based on Dairy Data [28] | ||||||||||||
End point description |
Percent change from EOT (up to Week 24) in EN caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
EOT (up to Week 24), EOS (up to Week 28)
|
||||||||||||
Notes [28] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Absolute Change From Baseline in Number of Hours per Day of Parenteral Support (PS) Usage at End of Treatment (EOT) Based on Dairy Data [29] | ||||||||||||
End point description |
Absolute change from baseline in number of hours per day of PS Usage at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT (up to Week 24)
|
||||||||||||
Notes [29] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Absolute Change From Baseline in Number of Days per Week of Parenteral Support (PS) Usage at End of Treatment (EOT) Based on Dairy Data [30] | ||||||||||||
End point description |
Absolute change from baseline in number of days per Week of PS usage at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. ITT population consisted of all subjects who were enrolled into the study and met all eligible criteria for this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT (up to Week 24)
|
||||||||||||
Notes [30] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
||||||||||
End point title |
Number of Subjects With Treatment-emergent Adverse Events (TEAEs) [31] | |||||||||
End point description |
An Adverse Event (AE) was any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. TEAEs were defined as any AEs whose onset occurred, severity worsened, or intensity increased after receiving the investigational product. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
|||||||||
End point type |
Primary
|
|||||||||
End point timeframe |
From start of study drug administration up to EOS (up to Week 28)
|
|||||||||
Notes [31] - 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: No statistical and comparison analyses were performed for this endpoint. |
||||||||||
|
||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Change From Baseline in Body Weight for age Z-score at Week 28 [32] | ||||||||||||
End point description |
Body weight was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in body weight for age Z-score at Week 28 was reported. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, Week 28
|
||||||||||||
Notes [32] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Change From Baseline in Height for Age Z-score at Week 28 [33] | ||||||||||||
End point description |
Height was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in height for age Z-score at Week 28 was reported. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, Week 28
|
||||||||||||
Notes [33] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Change From Baseline in Head Circumference for Age Z-score at Week 28 [34] | ||||||||||||
End point description |
Head circumference was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in head circumference for age Z-score at Week 28 was reported. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, Week 28
|
||||||||||||
Notes [34] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [35] - Data for this endpoint was not planned to be collected and analysed for Total Children. |
|||||||||||||
No statistical analyses for this end point |
|
||||||||||
End point title |
Number of Subjects With Clinically Significant Changes in Vital Signs Reported as Treatment Emergent Adverse Events (TEAEs) [36] | |||||||||
End point description |
Vital sign assessments included pulse rate, blood pressure, or body temperature. Number of subjects with clinically significant changes in vital signs by the investigator were recorded as TEAEs. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
|||||||||
End point type |
Primary
|
|||||||||
End point timeframe |
From start of study drug administration up to EOS (up to Week 28)
|
|||||||||
Notes [36] - 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: No statistical and comparison analyses were performed for this endpoint. |
||||||||||
|
||||||||||
No statistical analyses for this end point |
|
||||||||||
End point title |
Number of Subjects With Clinically Significant Changes in Electrocardiogram (ECG) Reported as Treatment Emergent Adverse Events (TEAEs) [37] | |||||||||
End point description |
12-lead ECG was performed. Any change in ECG assessments which were deemed to be clinically significant changes were recorded as TEAEs. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
|||||||||
End point type |
Primary
|
|||||||||
End point timeframe |
From start of study drug administration up to EOS (up to Week 28)
|
|||||||||
Notes [37] - 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: No statistical and comparison analyses were performed for this endpoint. |
||||||||||
|
||||||||||
No statistical analyses for this end point |
|
||||||||||
End point title |
Number of Subjects With Clinically Significant Laboratory Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) [38] | |||||||||
End point description |
Clinical laboratory assessments included biochemistry, hematology, coagulation, urinalysis. The number of subjects with clinically significant laboratory abnormalities were reported as TEAEs. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
|||||||||
End point type |
Primary
|
|||||||||
End point timeframe |
From start of study drug administration up to EOS (up to Week 28)
|
|||||||||
Notes [38] - 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: No statistical and comparison analyses were performed for this endpoint. |
||||||||||
|
||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Change From Baseline in the Average Urine Output at Week 28 [39] | ||||||||||||
End point description |
Average urine output was recorded in measured volume at Week 28 was reported. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed for the specified arm.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, Week 28
|
||||||||||||
Notes [39] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Change From Baseline in the Fecal Output at Week 28 [40] | ||||||||||||
End point description |
Change from baseline in the fecal output (Average number of stools per day) at Week 28 was recorded. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed for the specified arm.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, Week 28
|
||||||||||||
Notes [40] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
||||||||||
End point title |
Number of Subjects With Positive Specific Antibodies to Teduglutide [41] | |||||||||
End point description |
Number of subjects with positive specific antibodies to teduglutide were used to summarize the presence of antibodies. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
|||||||||
End point type |
Primary
|
|||||||||
End point timeframe |
From start of study drug administration up to EOS (up to Week 28)
|
|||||||||
Notes [41] - 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: No statistical and comparison analyses were performed for this endpoint. |
||||||||||
|
||||||||||
No statistical analyses for this end point |
|
||||||||||
End point title |
Number of Subjects With Clinically Significant Abnormal Findings in Gastrointestinal (GI) Specific Testing [42] | |||||||||
End point description |
GI specific testing included colonoscopy or sigmoidoscopy, abdominal ultrasound, fecal occult blood testing, upper GI series with small bowel follow-through (UGI/SBFT). EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Number of subjects with clinically significant abnormal findings in gastrointestinal specific testing were reported. Safety population consisted of all subjects in the ITT population who received at least 1 administration of investigational product with any safety follow-up.
|
|||||||||
End point type |
Primary
|
|||||||||
End point timeframe |
Baseline, EOT (up to Week 24)
|
|||||||||
Notes [42] - 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: No statistical and comparison analyses were performed for this endpoint. |
||||||||||
|
||||||||||
Notes [43] - Data for this endpoint was not planned to be collected and analysed for infants. |
||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Area Under the Concentration-time Curve at Steady State (AUCtau,ss) of Teduglutide in Plasma [44] | ||||||||||||
End point description |
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analysed using this study samples. Therefore, no PK parameters were reported in this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline: Pre-dose, 1, 6 hours post-dose; Week 4: Pre-dose, 2, 4 hours post-dose
|
||||||||||||
Notes [44] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [45] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. [46] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Maximum Plasma Concentration at Steady-state (Cmax,ss) of Teduglutide in Plasma [47] | ||||||||||||
End point description |
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analysed using this study samples. Therefore, no PK parameters were reported in this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline: Pre-dose, 1, 6 hours post-dose; Week 4: Pre-dose, 2, 4 hours post-dose
|
||||||||||||
Notes [47] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [48] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. [49] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Minimum Plasma Concentration at Steady-state (Cmin.ss) of Teduglutide in Plasma [50] | ||||||||||||
End point description |
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analysed using this study samples. Therefore, no PK parameters were reported in this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline: Pre-dose, 1, 6 hours post-dose; Week 4: Pre-dose, 2, 4 hours post-dose
|
||||||||||||
Notes [50] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [51] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. [52] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Time to Reach Maximum Observed Drug Concentration (Tmax) of Teduglutide in Plasma [53] | ||||||||||||
End point description |
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analysed using this study samples. Therefore, no PK parameters were reported in this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline: Pre-dose, 1, 6 hours post-dose; Week 4: Pre-dose, 2, 4 hours post-dose
|
||||||||||||
Notes [53] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [54] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. [55] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Terminal-Phase Half-life (t1/2) of Teduglutide in Plasma [56] | ||||||||||||
End point description |
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analysed using this study samples. Therefore, no PK parameters were reported in this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline: Pre-dose, 1, 6 hours post-dose; Week 4: Pre-dose, 2, 4 hours post-dose
|
||||||||||||
Notes [56] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [57] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. [58] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Apparent Clearance (CL/F) of Teduglutide [59] | ||||||||||||
End point description |
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analysed using this study samples. Therefore, no PK parameters were reported in this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline: Pre-dose, 1, 6 hours post-dose; Week 4: Pre-dose, 2, 4 hours post-dose
|
||||||||||||
Notes [59] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [60] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. [61] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
Apparent Volume of Distribution (V[lambda z]/F) of Teduglutide [62] | ||||||||||||
End point description |
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analysed using this study samples. Therefore, no PK parameters were reported in this study.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline: Pre-dose, 1, 6 hours post-dose; Week 4: Pre-dose, 2, 4 hours post-dose
|
||||||||||||
Notes [62] - 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: No statistical and comparison analyses were performed for this endpoint. |
|||||||||||||
|
|||||||||||||
Notes [63] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. [64] - PK was planned to be reported with other clinical studies. Hence,no individual PK data was reported. |
|||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Adverse events information
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
From start of study drug administration up to end of the study (up to Week 28)
|
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Assessment type |
Non-systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
20.0
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Reporting groups
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Reporting group title |
Total Children (1 - 15 years)
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Reporting group description |
Subjects with aged from 1 through 15 years received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Infants (4 - 12 months)
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Reporting group description |
Subjects with Infants aged from 4 through less than 12 months received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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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27 Apr 2016 |
Amendment 1:
- Identification of age range (1 through 15 years old).
- Update of severity of AEs (Utility of severity criteria of AE from National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) to evaluate dose interruption).
- Addition of continuation of output diary data collection over a 48-hour period of PS and enteral nutrition (EN) stability before every site visit. |
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06 Jun 2017 |
Amendment 2:
- Change of examination (removal of urine osmolality and urine sodium from the urinalysis). |
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24 Jan 2018 |
Amendment 3:
- Addition of direct bilirubin in laboratory tests. |
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12 Jun 2018 |
Amendment 4:
- Addition of infants with 4 months through <12 months corrected gestational age. |
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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 |