Clinical Trial Results:
A Prospective, Open-Label, Long-Term Safety and Efficacy Study of Teduglutide in Pediatric Patients With Short Bowel Syndrome Who Completed TED-C14-006 or SHP633-301
|
Summary
|
|
EudraCT number |
2016-000849-30 |
Trial protocol |
BE FI IT GB FR |
Global end of trial date |
05 Nov 2020
|
|
Results information
|
|
Results version number |
v1(current) |
This version publication date |
16 May 2021
|
First version publication date |
16 May 2021
|
Other versions |
|
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
|
|||
|
Trial identification
|
|||
Sponsor protocol code |
SHP633-304
|
||
|
Additional study identifiers
|
|||
ISRCTN number |
- | ||
US NCT number |
NCT02954458 | ||
WHO universal trial number (UTN) |
- | ||
|
Sponsors
|
|||
Sponsor organisation name |
Shire
|
||
Sponsor organisation address |
300 ShireWay, Lexington, United States, MA 02421
|
||
Public contact |
Study Director, Shire, +1 866 842 5335, ClinicalTransparency@takeda.com
|
||
Scientific contact |
Study Director, Shire, +1 866 842 5335, ClinicalTransparency@takeda.com
|
||
|
Paediatric regulatory details
|
|||
Is trial part of an agreed paediatric investigation plan (PIP) |
No
|
||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
|
Results analysis stage
|
|||
Analysis stage |
Final
|
||
Date of interim/final analysis |
05 Nov 2020
|
||
Is this the analysis of the primary completion data? |
No
|
||
Global end of trial reached? |
Yes
|
||
Global end of trial date |
05 Nov 2020
|
||
Was the trial ended prematurely? |
No
|
||
|
General information about the trial
|
|||
Main objective of the trial |
The primary objective of this study was to evaluate the long-term safety and tolerability of teduglutide treatment in pediatric subjects with short bowel syndrome (SBS) who completed their core study.
|
||
Protection of trial subjects |
The study was conducted in accordance with the ethical principles that have their origins in the Declaration of Helsinki and in compliance with all applicable industry regulations, International Council for Harmonisation (ICH) Good Clinical Practice Guideline E6 (1996), European Union (EU) Directive 2001/20/EC, as well as all applicable national and local laws and regulations.
|
||
Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
09 Jan 2017
|
||
Long term follow-up planned |
No
|
||
Independent data monitoring committee (IDMC) involvement? |
Yes
|
||
|
Population of trial subjects
|
|||
Number of subjects enrolled per country |
|||
Country: Number of subjects enrolled |
Belgium: 1
|
||
Country: Number of subjects enrolled |
Canada: 4
|
||
Country: Number of subjects enrolled |
Finland: 4
|
||
Country: Number of subjects enrolled |
United Kingdom: 12
|
||
Country: Number of subjects enrolled |
Italy: 1
|
||
Country: Number of subjects enrolled |
United States: 39
|
||
Worldwide total number of subjects |
61
|
||
EEA total number of subjects |
6
|
||
Number of subjects enrolled per age group |
|||
In utero |
0
|
||
Preterm newborn - gestational age < 37 wk |
0
|
||
Newborns (0-27 days) |
0
|
||
Infants and toddlers (28 days-23 months) |
6
|
||
Children (2-11 years) |
51
|
||
Adolescents (12-17 years) |
3
|
||
Adults (18-64 years) |
1
|
||
From 65 to 84 years |
0
|
||
85 years and over |
0
|
||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
|
Recruitment
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
Recruitment details |
The study was conducted at 23 sites across the United States, Belgium, Canada, United Kingdom, Finland, and Italy between 09 January 2017 (first subject first visit) and 05 Nov 2020 (last subject last visit). | ||||||||||||||||||||||||||||||||||||||||||||||||||
|
Pre-assignment
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
Screening details |
Total of 61 subjects (children and infant) were enrolled in 4 groups: Non-teduglutide/Non-teduglutide(NTT/NTT), Non-teduglutide/Teduglutide(NTT/TED), Teduglutide /Non-teduglutide(TED/NTT) and Teduglutide/Teduglutide(TED/TED). Subjects who completed core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were eligible for this study. | ||||||||||||||||||||||||||||||||||||||||||||||||||
|
Period 1
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
Period 1 title |
Overall Study (overall period)
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Is this the baseline period? |
Yes | ||||||||||||||||||||||||||||||||||||||||||||||||||
Allocation method |
Not applicable
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Blinding used |
Not blinded | ||||||||||||||||||||||||||||||||||||||||||||||||||
|
Arms
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
Are arms mutually exclusive |
Yes
|
||||||||||||||||||||||||||||||||||||||||||||||||||
|
Arm title
|
Non-teduglutide/Non-teduglutide Treatment (NTT/NTT) | ||||||||||||||||||||||||||||||||||||||||||||||||||
Arm description |
Subjects who participated in standard of care arm in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study but did not receive any teduglutide treatment in no-teduglutide period up to 39 months (Each visit =12 weeks of cycle up to 14 visits) in the study. | ||||||||||||||||||||||||||||||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product name |
Teduglutide
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product code |
SHP633
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Other name |
|||||||||||||||||||||||||||||||||||||||||||||||||||
Pharmaceutical forms |
Powder and solvent for solution for injection
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Routes of administration |
Subcutaneous use
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Dosage and administration details |
Subjects who participated in standard of care arm in the core study and did not receive any teduglutide treatment in the this extension study.
|
||||||||||||||||||||||||||||||||||||||||||||||||||
|
Arm title
|
Non-teduglutide/Teduglutide Treatment (NTT/TED) | ||||||||||||||||||||||||||||||||||||||||||||||||||
Arm description |
Subjects who participated in standard of care arm in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study and received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injections once daily up to 36 months (Up to 6 Cycles [each Cycle 1 to 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks]). | ||||||||||||||||||||||||||||||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product name |
Teduglutide
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product code |
SHP633
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Other name |
|||||||||||||||||||||||||||||||||||||||||||||||||||
Pharmaceutical forms |
Powder and solvent for solution for injection
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Routes of administration |
Subcutaneous use
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Dosage and administration details |
Subjects who participated in standard of care arm in the core study and received 0.05 mg/kg teduglutide once daily in this extension study.
|
||||||||||||||||||||||||||||||||||||||||||||||||||
|
Arm title
|
Teduglutide /Non-teduglutide Treatment (TED/NTT) | ||||||||||||||||||||||||||||||||||||||||||||||||||
Arm description |
Subjects who received teduglutide in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study but did not receive any teduglutide treatment in no-teduglutide period up to 39 months (Each visit =12 weeks of cycle up to 14 visits) in the study. | ||||||||||||||||||||||||||||||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product name |
Teduglutide
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product code |
SHP633
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Other name |
|||||||||||||||||||||||||||||||||||||||||||||||||||
Pharmaceutical forms |
Powder and solvent for solution for injection
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Routes of administration |
Subcutaneous use
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Dosage and administration details |
Subjects received 0.05 mg/kg teduglutide in the core study but not in the this extension study;
|
||||||||||||||||||||||||||||||||||||||||||||||||||
|
Arm title
|
Teduglutide/Teduglutide Treatment (TED/TED) | ||||||||||||||||||||||||||||||||||||||||||||||||||
Arm description |
Subjects who received teduglutide in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study received 0.05 mg/kg of teduglutide SC injections once daily up to 36 months (Up to 6 Cycles [each Cycle 1 to 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks]). | ||||||||||||||||||||||||||||||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product name |
Teduglutide
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Investigational medicinal product code |
SHP633
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Other name |
|||||||||||||||||||||||||||||||||||||||||||||||||||
Pharmaceutical forms |
Powder and solvent for solution for injection
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Routes of administration |
Subcutaneous use
|
||||||||||||||||||||||||||||||||||||||||||||||||||
Dosage and administration details |
Subjects received 0.05 mg/kg teduglutide once daily in the core study and in this extension study.
|
||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Baseline characteristics reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Non-teduglutide/Non-teduglutide Treatment (NTT/NTT)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects who participated in standard of care arm in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study but did not receive any teduglutide treatment in no-teduglutide period up to 39 months (Each visit =12 weeks of cycle up to 14 visits) in the study. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Non-teduglutide/Teduglutide Treatment (NTT/TED)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects who participated in standard of care arm in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study and received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injections once daily up to 36 months (Up to 6 Cycles [each Cycle 1 to 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks]). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Teduglutide /Non-teduglutide Treatment (TED/NTT)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects who received teduglutide in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study but did not receive any teduglutide treatment in no-teduglutide period up to 39 months (Each visit =12 weeks of cycle up to 14 visits) in the study. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Teduglutide/Teduglutide Treatment (TED/TED)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects who received teduglutide in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study received 0.05 mg/kg of teduglutide SC injections once daily up to 36 months (Up to 6 Cycles [each Cycle 1 to 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks]). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||
|
End points reporting groups
|
|||
Reporting group title |
Non-teduglutide/Non-teduglutide Treatment (NTT/NTT)
|
||
Reporting group description |
Subjects who participated in standard of care arm in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study but did not receive any teduglutide treatment in no-teduglutide period up to 39 months (Each visit =12 weeks of cycle up to 14 visits) in the study. | ||
Reporting group title |
Non-teduglutide/Teduglutide Treatment (NTT/TED)
|
||
Reporting group description |
Subjects who participated in standard of care arm in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study and received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injections once daily up to 36 months (Up to 6 Cycles [each Cycle 1 to 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks]). | ||
Reporting group title |
Teduglutide /Non-teduglutide Treatment (TED/NTT)
|
||
Reporting group description |
Subjects who received teduglutide in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study but did not receive any teduglutide treatment in no-teduglutide period up to 39 months (Each visit =12 weeks of cycle up to 14 visits) in the study. | ||
Reporting group title |
Teduglutide/Teduglutide Treatment (TED/TED)
|
||
Reporting group description |
Subjects who received teduglutide in the core study TED-C14-006 [2015-002252-27] or SHP633-301 [2017-003606-40] were enrolled into this extension study received 0.05 mg/kg of teduglutide SC injections once daily up to 36 months (Up to 6 Cycles [each Cycle 1 to 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks]). | ||
|
||||||||||||||||
End point title |
Number of Subjects With Treatment-emergent Adverse Events (TEAEs) [1] | |||||||||||||||
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 AEs that started or worsened on or after the date of first dose of teduglutide for subjects in the TED/TED, TED/NTT, and NTT/TED treatment groups, or after the core study baseline visit for subjects in the NTT/NTT group. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria.
|
|||||||||||||||
End point type |
Primary
|
|||||||||||||||
End point timeframe |
From start of study drug administration up to follow-up (up to Month 46)
|
|||||||||||||||
| 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 comparision analyses were performed for this endpoint. |
||||||||||||||||
|
||||||||||||||||
| No statistical analyses for this end point | ||||||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Average Total Urine Output at End of Treatment (EOT) of Last Cycle During Teduglutide Treatment Period [2] [3] | ||||||||||||
End point description |
Average total urine output was recorded over a 48-hour period of parental support (PS) stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The Average daily urine output milliliter per kilogram per day (mL/kg/day) was calculated as: Total urine output over 48 hours / 2) / body weight (kilogram [kg]) where total urine output was calculated as the sum of the urine output in milliliter (mL) and the urine-only diaper weights in gram (g) (1g = 1mL) for the subject collected on the output diary form of electronic case report from (eCRF). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| 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 comparision analyses were performed for this endpoint. [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline in Average Total Urine Output at Last Visit During Non-Teduglutide (NT) Period [4] | ||||||||||||||||||||
End point description |
Average total urine output was recorded over a 48 hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. Average daily urine output mL/kg/day was calculated as: (Total urine output over 48 hours / 2) / body weight (kg) where total urine output was calculated as the sum of the urine output in mL and the urine-only diaper weights in gram (1g = 1mL) for the subject collected on the output diary form of electronic case report from (eCRF). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed 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, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| Notes [5] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Average Number of Stools per Day at EOT of Last Cycle During Teduglutide Treatment Period [6] [7] | ||||||||||||
End point description |
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized by the average number of stools per day. The average number of stools per day was calculated as (sum of the daily data in a 48-hour period/2).The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| Notes [6] - 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 comparision analyses were performed for this endpoint. [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| Notes [8] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline in Average Number of Stools per Day at Last Visit During Non-Teduglutide Period [9] | ||||||||||||||||||||
End point description |
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized by the average number of stools per day. The average number of stools per day was calculated as (sum of the daily data in a 48-hour period/2). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint.
|
||||||||||||||||||||
End point type |
Primary
|
||||||||||||||||||||
End point timeframe |
Baseline, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| Notes [10] - Data was not collected and analysed at this time point, for this respective arm. [11] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Average Total Daily Stool/Mixed Stool Diaper Weight at EOT of Last Cycle During Teduglutide Treatment Period [12] [13] | ||||||||||||
End point description |
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized by the average total daily stool/mixed stool diaper weight (gram per kilogram per day [g/kg/day]). The body weight was used to calculate the daily stool/mixed stool diaper weight (g/kg/day). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed 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. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| 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 comparision analyses were performed for this endpoint. [13] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change from Baseline in Average Total Daily Stool/Mixed Stool Diaper Weight at Last Visit During Non-Teduglutide Period [14] | ||||||||||||||||||||
End point description |
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized by the average total daily stool/mixed stool diaper weight (g/kg/day). The body weight was used to calculate the daily stool/mixed stool diaper weight (g/kg/day). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed 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, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| Notes [15] - Data was not collected and analysed at this time point, for this respective arm. [16] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Average Total Ostomy Output at EOT of Last Cycle During Teduglutide Treatment Period [17] [18] | ||||||||||||
End point description |
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized the average ostomy output per day (mL/kg/day). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| 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 comparision analyses were performed for this endpoint. [18] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline in Average Total Ostomy Output at Last Visit During Non-Teduglutide Period [19] | ||||||||||||||||||||
End point description |
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized the average ostomy output per day. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint.
|
||||||||||||||||||||
End point type |
Primary
|
||||||||||||||||||||
End point timeframe |
Baseline, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| Notes [20] - Data was not collected and analysed at this time point, for this respective arm. [21] - Data was not collected and analysed at this time point, for this respective arm. [22] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Average Bristol Stool Form Score at EOT of Last Cycle During Teduglutide Treatment Period [23] [24] | ||||||||||||
End point description |
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. Average daily fecal output was summarized the average typical stool form score using Bristol Stool Form Scale. Average typical stool form score was calculated as (sum of the daily data in a 48-hour period / 2). Typical Stool Form based on Bristol Stool Form Scale: 1 - Separate hard lumps, hard to pass, 2 - Sausage-shaped, but lumpy, 3 - Like a sausage but with cracks on the surface, 4- Like a sausage or snake, smooth and soft, 5- Soft blobs with clear-cut edges, 6- Fluffy pieces with ragged edges, a mushy stool, 7- Watery, no solid pieces. Entirely liquid. Analyis was planned based on safety population. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| 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 comparision analyses were performed for this endpoint. [24] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| Notes [25] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline in Average Bristol Stool Form Score at Last Visit During Non-Teduglutide Period [26] | ||||||||||||||||||||
End point description |
Fecal output was recorded over a 48 hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. Average daily fecal output was summarized separately the average typical stool form score using Bristol Stool Form Scale. Average typical stool form score was calculated as (sum of the daily data in a 48-hour period / 2). Typical Stool Form based on Bristol Stool Form Scale: 1- Separate hard lumps, hard to pass, 2- Sausage-shaped, but lumpy, 3- Like a sausage but with cracks on the surface, 4 - Like a sausage or snake, smooth and soft, 5- Soft blobs with clear-cut edges, 6- Fluffy pieces with ragged edges, a mushy stool, 7- Watery, no solid pieces entirely liquid. Analysis were performed by safety population. Number of subjects analysed refer to the subjects evaluable for this endpoint. Here, '99999' indicates standard deviation was not estimated as only single subject was analysed.
|
||||||||||||||||||||
End point type |
Primary
|
||||||||||||||||||||
End point timeframe |
Baseline, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| Notes [27] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||
End point title |
Number of Subjects With Positive Specific Antibodies [28] [29] | ||||||||||||
End point description |
Number of subjects with positive specific antibodies to teduglutide were used to summarize the presence of antibodies. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, TED/NTT and TED/TED arms only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline up to EOS (Up to Month 46)
|
||||||||||||
| 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 comparision analyses were performed for this endpoint. [29] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Body Weight Z-score at EOT of Last Cycle During Teduglutide Treatment Period [30] [31] | ||||||||||||
End point description |
Body weight was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts are used for calculation. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| 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 comparision analyses were performed for this endpoint. [31] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline in Body Weight Z-score at Last Visit During Non-Teduglutide Period [32] | ||||||||||||||||||||
End point description |
Body weight was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts are used for calculation. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed 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. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||
End point type |
Primary
|
||||||||||||||||||||
End point timeframe |
Baseline, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Height Z-score at EOT of Last Cycle During Teduglutide Treatment Period [33] [34] | ||||||||||||
End point description |
Height was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts are used for calculation. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| 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 comparision analyses were performed for this endpoint. [34] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline in Height Z-score at Last Visit During Non-Teduglutide Period [35] | ||||||||||||||||||||
End point description |
Height was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts are used for calculation. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed 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, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| Notes [35] - 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Head Circumference Z-score at EOT of Last Cycle During Teduglutide Treatment Period [36] [37] | ||||||||||||
End point description |
Head circumference was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts are used for calculation. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| 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 comparision analyses were performed for this endpoint. [37] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| Notes [38] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline in Head Circumference Z-score at Last Visit During Non-Teduglutide Period [39] | ||||||||||||||||||||
End point description |
Head circumference was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts are used for calculation. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed 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, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| Notes [40] - Data was not collected and analysed at this time point, for this respective arm. [41] - Data was not collected and analysed at this time point, for this respective arm. [42] - Data was not collected and analysed at this time point, for this respective arm. |
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||
End point title |
Change From Baseline in Body Mass Index (BMI) Z-score at EOT of Last Cycle During Teduglutide Treatment Period [43] [44] | ||||||||||||
End point description |
BMI Z-score was calculated by using the height and weight data. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts are used for calculation. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed refer to the subjects evaluable for this endpoint. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||
End point type |
Primary
|
||||||||||||
End point timeframe |
Baseline, EOT of last cycle (Up to Month 36) (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||
| Notes [43] - 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 comparision analyses were performed for this endpoint. [44] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline in Body Mass Index (BMI) Z-score at Last Visit During Non-Teduglutide Period [45] | ||||||||||||||||||||
End point description |
BMI Z-score was calculated by using the height and weight data. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts are used for calculation. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analyzed 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, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
| Notes [45] - 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 comparision analyses were performed for this endpoint. |
|||||||||||||||||||||
|
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Number of Subjects Who Achieved At least 20 Percent (%), 50%, and 75% Reduction From Baseline in Diary Parenteral Support (PS) Volume at End of Treatment (EOT) of Each Cycle During Teduglutide Treatment Period [46] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Number of subjects who achieved at least 20%, 50%, and 75% reduction from baseline in diary PS volume at EOT of last cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, "n=number analysed" refer to subjects who were evaluable at given categories. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Notes [46] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| No statistical analyses for this end point | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Number of Subjects Who Achieved At least 20 Percent (%), 50%, and 75% Reduction From Baseline in Prescribed PS Volume at End of Treatment (EOT) of Each Cycle During Teduglutide Treatment Period [47] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Number of subjects who achieved at least 20%, 50%, and 75% reduction from baseline in prescribed PS volume at EOT of east cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, "n=number analysed" refer to subjects who were evaluable at given categories. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Notes [47] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| No statistical analyses for this end point | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment Period [48] | ||||||||||||||||||||||||||||||
End point description |
Change from baseline in diary PS volume at EOT of each cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [48] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Percent Change From Baseline in Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment Period [49] | ||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in diary PS volume at EOT of each cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [49] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment Period [50] | ||||||||||||||||||||||||||||||
End point description |
Change from baseline in prescribed PS volume at EOT of each cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, "n=number analysed" refer to subjects who were evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [50] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Percent Change From Baseline in Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment Period [51] | ||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in prescribed PS volume at EOT of each cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, "n=number analysed" refer to subjects who were evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [51] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Diary PS Caloric Intake at EOT of Each Cycle During Teduglutide Treatment Period [52] | ||||||||||||||||||||||||||||||
End point description |
Change from baseline in diary PS caloric intake at EOT of each cycle during teduglutide treatment period was reported. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [52] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Percent Change From Baseline in Diary PS Caloric Intake at EOT of Each Cycle During Teduglutide Treatment Period [53] | ||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in diary PS Caloric intake at EOT of each cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [53] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Prescribed PS Caloric Intake at EOT of Each Cycle During Teduglutide Treatment Period [54] | ||||||||||||||||||||||||||||||
End point description |
Change from baseline in prescribed PS caloric intake at EOT of each cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [54] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Percent Change From Baseline in Prescribed PS Caloric Intake at EOT of Each Cycle During Teduglutide Treatment Period [55] | ||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in prescribed PS caloric intake at EOT of each cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [55] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||
End point title |
Number of Subjects Who Achieved 100% Reduction in Complete Weaning of PS Volume at EOT of Each Cycle During Teduglutide Treatment Period [56] | |||||||||||||||||||||||||||
End point description |
Number of subjects who achieved at least 100% reduction in complete weaning of PS volume at EOT of each cycle during teduglutide treatment period was reported. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, "n=number analysed" refer to subjects who were evaluable at given categories. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
|||||||||||||||||||||||||||
End point type |
Secondary
|
|||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
|||||||||||||||||||||||||||
| Notes [56] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||
| No statistical analyses for this end point | ||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Hours per Day of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment Period [57] | ||||||||||||||||||||||||||||||
End point description |
Change from baseline in hours per day of diary PS usage at EOT of each cycle during teduglutide treatment period was reported. Hours per day of diary PS was calculated as: Hours per day of actual PS = (sum of hours per day for each day that PS intake data is recorded within the 7 days prior to the visit / number of days that PS hours per day data is recorded as non-zero within the 7 days prior to the visit). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [57] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Percent Change From Baseline in Hours per Day of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment Period [58] | ||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in hours per day of diary PS usage at EOT of each cycle during teduglutide treatment period was reported. Hours per day of diary PS was calculated as: Hours per day of actual PS = (sum of hours per day for each day that PS intake data is recorded within the 7 days prior to the visit / number of days that PS hours per day data is recorded as non-zero within the 7 days prior to the visit). Safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [58] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Hours per Day of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment Period [59] | ||||||||||||||||||||||||||||||
End point description |
Change from baseline in hours per day of prescribed PS usage at EOT of each cycle during teduglutide treatment period was reported. Hours per day of prescribed PS was calculated as: Hours per day of actual PS = (sum of hours per day for each day that PS intake data is recorded within the 7 days prior to the visit / number of days that PS hours per day data is recorded as non-zero within the 7 days prior to the visit). The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, "n=number analysed" refer to subjects who were evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [59] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Percent Change From Baseline in Hours per Day of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment Period [60] | ||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in hours per day of prescribed PS usage at EOT of each cycle during teduglutide treatment period was reported. Hours per day of prescribed PS was calculated as: Hours per day of actual PS = (sum of hours per day for each day that PS intake data is recorded within the 7 days prior to the visit / number of days that PS hours per day data is recorded as non-zero within the 7 days prior to the visit). Safety population; all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [60] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Days per Week of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment Period [61] | ||||||||||||||||||||||||||||||
End point description |
Change from baseline in days per week of diary PS usage at EOT of each cycle during teduglutide treatment period was reported. Days per week of diary PS was calculated as: Days per week of actual PS = (number of days with non-zero values for PS volume within the 7 days prior to the visit / number of days for which any PS intake data was recorded within the 7 days prior to the visit) * 7. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [61] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Percent Change From Baseline in Days per Week of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment Period [62] | ||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in days per week of diary PS usage at EOT of each cycle during teduglutide treatment period was reported. Days per week of diary PS was calculated as: Days per week of actual PS = (number of days with non-zero values for PS volume within the 7 days prior to the visit / number of days for which any PS intake data was recorded within the 7 days prior to the visit) * 7. Safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [62] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline in Days per Week of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment Period [63] | ||||||||||||||||||||||||||||||
End point description |
Change from baseline in days per week of prescribed PS usage at EOT of each cycle during teduglutide treatment period was reported. Days per week of prescribed PS was calculated as: Days per week of actual PS = (number of days with non-zero values for PS volume within the 7 days prior to the visit / number of days for which any PS intake data was recorded within the 7 days prior to the visit) * 7. The safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, "n=number analysed" refer to subjects who were evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [63] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Percent Change From Baseline in Days per Week of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment Period [64] | ||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in days per week of prescribed PS usage at EOT of each cycle during teduglutide treatment period was reported. Days per week of prescribed PS was calculated as: Days per week of actual PS = (number of days with non-zero values for PS volume within the 7 days prior to the visit / number of days for which any PS intake data was recorded within the 7 days prior to the visit) * 7. Safety population consisted all enrolled subjects who provided informed consent and met all the inclusion criteria. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [64] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Score at EOT of Each Cycle During Teduglutide Treatment Period [65] | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
PedsQL GCS was designed to measure health-related quality of life in pediatric subjects and adolescents (2 to 18 years of age). It encompasses 4 dimensions of functioning (physical [8 items], emotional [5 items], social [5 items], school [3 items]). Age groups were: Toddler (2-4 years), Young pediatric (5-7 years), Pediatric (8-12 years), and Teens (13-18 years). Depending on the subjects age, the questionnaire may be completed by parent/caregiver as appropriate. For Toddler group, PedsQL GCS consisted of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, PedsQL GCS consisted of 23 items, with a 3-point Likert scale (0, 2, 4) for the young pediatric, and a 5-point Likert scale for the pediatric and teens groups. Scores are transformed on a scale from 0 to 100 where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life. Analysis: safety population. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Other pre-specified
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Notes [65] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline In PedsQL GCS Score at Last Visit During Non-Teduglutide Period | ||||||||||||||||||||||||||||||||||||||||
End point description |
PedsQL GCS was designed to measure health-related quality of life in pediatric subjects and adolescents (2 to 18 years of age). It encompasses 4 dimensions of functioning (physical [8 items], emotional [5 items], social [5 items], school [3 items]). Age groups were: Toddler (2-4 years), Young pediatric (5-7 years), Pediatric (8-12 years), and Teens (13-18 years). Depending on the subject’s age, the questionnaire may be completed by parent/caregiver as appropriate. For the Toddler group, the PedsQL GCS consisted of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consisted of 23 items, with a 3-point Likert scale (0, 2, 4) for the young pediatric, and a 5-point Likert scale for the pediatric and teens groups. Scores are transformed on a scale from 0 to 100 where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life. Analysis: safety population. Here, "n=number analysed" refer to subjects evaluable at given categories.
|
||||||||||||||||||||||||||||||||||||||||
End point type |
Other pre-specified
|
||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Baseline, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline In PedsQL Total Family Impact Module Score at EOT of Each Cycle During Teduglutide Treatment Period [66] | ||||||||||||||||||||||||||||||
End point description |
PedsQL Family Impact Module was composed of 36 items comprising Physical Functioning (6 items), Emotional Functioning (5 items), Social Functioning (4 items), Cognitive Functioning (5 items), Communication (3 items), Worry (5 items), Daily Activities (3 items) and Family Relationships (5 items). Total family impact score was calculated as the sum of all the items over the number of items answered on all the scales. Scores are transformed on a scale from 0 to 100 were 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life. Analysis was performed based on safety population. Here, "n=number analysed" refer to subjects evaluable at given categories. Here, '99999' indicates that standard deviation was not estimated as only single subject was analysed at specified time point and '9999' indicates that data was not estimated as no subject was analysed at specified time point. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||
End point type |
Other pre-specified
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||
| Notes [66] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||
End point title |
Change From Baseline In PedsQL Total Family Impact Module Score at Last Visit During Non-Teduglutide Period | ||||||||||||||||||||
End point description |
PedsQL Family Impact Module was composed of 36 items comprising Physical Functioning (6 items), Emotional Functioning (5 items), Social Functioning (4 items), Cognitive Functioning (5 items), Communication (3 items), Worry (5 items), Daily Activities (3 items) and Family Relationships (5 items). Total family impact score was calculated as the sum of all the items over the number of items answered on all the scales. Scores are transformed on a scale from 0 to 100 were 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life. Analysis was performed based on safety population. Here, 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 |
Other pre-specified
|
||||||||||||||||||||
End point timeframe |
Baseline, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||
|
|||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||
End point title |
Change From Baseline In PedsQL Gastrointestinal Symptoms Module at EOT of Each Cycle During Teduglutide Treatment Period [67] | ||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
PedsQL Gastrointestinal symptoms module was composed of 58 items, comprised of 10 different symptom scales that assess gastrointestinal symptom-related quality of life: food and drink limits, trouble swallowing, heartburn and reflux, nausea and vomiting, gas and bloating, constipation, blood in poop, and diarrhea. Only the scales of food and drink limits (6 items) and diarrhea (7 items) was used in this study. Sub-scale score was calculated as the sum of the items over the number of items answered in the scale. Scores are transformed on a scale from 0 to 100 were 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life. Analysis was performed based on safety population. Here, number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. Data was planned to be collected and analysed for NTT/TED, and TED/TED arm only.
|
||||||||||||||||||||||||||||||||||||||||||||||||
End point type |
Other pre-specified
|
||||||||||||||||||||||||||||||||||||||||||||||||
End point timeframe |
Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks)
|
||||||||||||||||||||||||||||||||||||||||||||||||
| Notes [67] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” treatment period only. |
|||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
End point title |
Change From Baseline In PedsQL Gastrointestinal Symptoms Module at Last Visit During Non-Teduglutide Period | ||||||||||||||||||||||||||||||
End point description |
PedsQL gastrointestinal symptoms module was composed of 58 items, comprised of 10 different symptom scales that assess gastrointestinal symptom-related quality of life: food and drink limits, trouble swallowing, heartburn and reflux, nausea and vomiting, gas and bloating, constipation, blood in poop, and diarrhea. Only the scales of food and drink limits (6 items) and diarrhea (7 items) was used in this study. Sub-scale score was calculated as the sum of the items over the number of items answered in the scale. Scores are transformed on a scale from 0 to 100 were 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life. Analysis: safety population. Number of subjects analysed refer to the subjects evaluable for this endpoint and "n=number analysed" refer to subjects evaluable at given categories. '99999' indicates standard deviation was not estimated as only single subject was analysed for the specified arm.
|
||||||||||||||||||||||||||||||
End point type |
Other pre-specified
|
||||||||||||||||||||||||||||||
End point timeframe |
Baseline, Last visit in NT (up to Month 39) (Each visit= 12 weeks)
|
||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Adverse events information
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
From start of study drug administration up to follow-up (up to Month 46)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assessment type |
Non-systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Dictionary used for adverse event reporting
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
19.1
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Non-teduglutide/Non-teduglutide Treatment (NTT/NTT)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects who participated in standard of care arm in the core study TED-C14-006 [NCT02682381] or SHP633-301 [NCT03571516] were enrolled into this extension study but did not receive any teduglutide treatment in no-teduglutide period up to 39 months (Each visit =12 weeks of cycle up to 14 visits) in the study. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Non-teduglutide/Teduglutide Treatment (NTT/TED)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects who participated in standard of care arm in the core study TED-C14-006 [NCT02682381] or SHP633-301 [NCT03571516] were enrolled into this extension study and received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injections once daily up to 36 months (Up to 6 Cycles [each Cycle 1 to 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks]). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Teduglutide /Non-teduglutide Treatment (TED/NTT)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects who received teduglutide in the core study TED-C14-006 [NCT02682381] or SHP633-301 [NCT03571516] were enrolled into this extension study but did not receive any teduglutide treatment in no-teduglutide period up to 39 months (Each visit =12 weeks of cycle up to 14 visits) in the study. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Teduglutide/Teduglutide Treatment (TED/TED)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects who received teduglutide in the core study TED-C14-006 [NCT02682381] or SHP633-301 [NCT03571516] were enrolled into this extension study received 0.05 mg/kg of teduglutide SC injections once daily up to 36 months (Up to 6 Cycles [each Cycle 1 to 4 = 28 weeks, Cycle 5 = 27 weeks, and Cycle 6 = 16 weeks]). | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||
Substantial protocol amendments (globally) |
|||
| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
||
22 Nov 2016 |
Protocol Amendment 1:
- The collection of all actual and prescribed EN data was removed to reduce the burden on the subjects and investigators. Enteral nutrition data are not required as the efficacy endpoints are limited to PS parameters.
- Parental height and gestational age at birth were removed from medical history.
- Changes were made to the Health economics and outcomes research endpoints to include the beginning of each treatment cycle (CxD1) as additional baseline. These changes were for clarity and consistency with other teduglutide studies. |
||
23 Mar 2017 |
Protocol Amendment 2:
- Revised the criteria for ET of the study: stopping criteria were extended to all NCI CTCAE Grade 3 and 4 severity events reported as related to the investigational product, and no longer limited to the events described in Table 8-1, titled “CTCAE Criteria for Adverse Events that May Lead to Dose Interruption (Prospective Period of Observation Only).” |
||
16 May 2018 |
Protocol Amendment 3:
- To minimize risk to subjects, a new escape criterion was added, allowing those who had escaped during the follow-up period of a previous teduglutide treatment to omit the follow-up period during subsequent teduglutide treatment cycles. For subjects who previously escaped the follow-up period, CxW24 assessments could be combined with the next pretreatment visit assessments.
- Updated the information on the clinical studies with teduglutide in pediatric subjects to include the results of TED-C14-006 and a description of the additional core study, SHP633-301.
- Added new PK simulation data to further support dosing.
- Specified that an interim analysis was planned when 6 months of safety data had been collected for subjects entering from TED-C14-006. Additional interim analyses could be conducted as needed. |
||
01 Oct 2019 |
Protocol Amendment 4:
- Extended the planned study period to December 2020.
- Clarification of the follow-up period escape criteria and their use in combining the CxW24 and pretreatment visits was added.
- Clarified that AEs will be collected for 4 weeks after the last dose of teduglutide in the study even if the EOS/ET occurs within that timeframe.
- The definition of an overdose was clarified as the administration of the investigational product at a dose or frequency greater than 0.05 mg/kg subcutaneous once daily. An overdose occurred if any of the following criteria were met:
- More than 0.05 mg/kg was given at any one time
- Consecutive doses were spaced less than 12 hours apart
- Any more than 0.05 mg/kg given in one day
- The teduglutide dose interruption criteria were updated to specify events leading to an interruption of teduglutide treatment and events leading to a permanent discontinuation of teduglutide treatment. Investigational product was interrupted if any of the following events occurred:
- An AE of special interest
- An AE that is of NCI CTCAE severity Grade 3 or 4 and related to teduglutide
- Intestinal obstruction
- Biliary obstruction related to teduglutide
- Pancreatic duct obstruction related to teduglutide
- Heart failure with severe fluid overload related to teduglutide.
- Investigational product was permanently discontinued if any of the following events occurred:
- Pregnancy
- Severe hypersensitivity, such as anaphylaxis determined by the investigator to be related to study drug. This does not include the presence of anti-teduglutide antibodies, mild injection site reactions or mild symptoms that according to the investigator do not pose a significant risk to the subject.
- Confirmed drug-induced liver injury (DILI) related to teduglutide.
- Any malignancy. |
||
Interruptions (globally) |
|||
| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
|||
| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
