Clinical Trial Results:
A Phase 3, Open-label Safety Study of Teduglutide in Japanese Pediatric Patients With Short Bowel Syndrome Who are Dependent on Parenteral Support, Aged 4 Months of Corrected Gestational Age or Older, and Requiring the Dosing of 1.25 mg Formulation
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Summary
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EudraCT number |
2022-003572-16 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
27 Sep 2023
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Results information
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Results version number |
v1(current) |
This version publication date |
05 Apr 2024
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First version publication date |
05 Apr 2024
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
TAK-633-3008
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT05027308 | ||
WHO universal trial number (UTN) |
U1111-1267-3327 | ||
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Sponsors
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Sponsor organisation name |
Takeda
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Sponsor organisation address |
95 Hayden Avenue, Lexington, United States, MA 02421
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Public contact |
Study Director, Takeda, TrialDisclosures@takeda.com
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Scientific contact |
Study Director, Takeda, TrialDisclosures@takeda.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
27 Sep 2023
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
27 Sep 2023
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The main objective of the trial was to check for side effects from teduglutide in Japanese Children With Short Bowel Syndrome.
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Protection of trial subjects |
All study participants were required to read and sign an Informed Consent Form.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
04 Jan 2022
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Japan: 3
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Worldwide total number of subjects |
3
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
2
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Children (2-11 years) |
1
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
Three participants took part in the study at six investigative sites in Japan from 4 January 2022 to 27 September 2023. | ||||||
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Pre-assignment
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Screening details |
Pediatric participants with a diagnosis of short bowel syndrome (SBS) dependent on parenteral support (PS) were enrolled in the study based on the eligibility criteria to receive teduglutide. | ||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
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Arms
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Arm title
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Teduglutide | ||||||
Arm description |
Participants received teduglutide 0.05 milligram per kilogram [mg/kg] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous [SC] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles. | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
Teduglutide
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Investigational medicinal product code |
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Other name |
TAK-633
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Pharmaceutical forms |
Injection
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
Teduglutide 0.05 mg/kg SC injection
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Baseline characteristics reporting groups
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Reporting group title |
Teduglutide
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Reporting group description |
Participants received teduglutide 0.05 milligram per kilogram [mg/kg] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous [SC] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Teduglutide
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Reporting group description |
Participants received teduglutide 0.05 milligram per kilogram [mg/kg] (0.025 mg/kg for participants with moderate or greater renal impairment) subcutaneous [SC] injection once daily in a 28-week treatment cycle consisting of a 24-week treatment period followed by a 4-week no treatment follow-up period for a maximum of 3 cycles. | ||
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End point title |
Number of Participants With Treatment-emergent Adverse Events (TEAEs) [1] | ||||||||
End point description |
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. TEAEs were defined as any AEs whose onset occurred, severity worsened, or intensity increased after receiving the investigational product. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
From first dose of study drug until follow-up visit (4 weeks after end of treatment [EOT]/end of termination [ET] {up to 47.3-51.3 weeks})
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Participants With Adverse Events of Special Interest (AESIs) [2] | ||||||||
End point description |
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AESI, whether serious or non-serious, is one of scientific and medical concern specific to the compound or program, for which ongoing monitoring and rapid communication by the investigator to the sponsor may be appropriate. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
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| Notes [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Participants With Serious Adverse Events (SAEs) [3] | ||||||||
End point description |
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An SAE is defined as any untoward medical occurrence that at any dose: results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to a congenital anomaly /birth defect, is the other important medical event. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
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| Notes [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Participants With Clinically Significant Abnormalities in Vital Signs Reported as an Adverse Event [4] | ||||||||
End point description |
Vital signs include systolic and diastolic blood pressure, heart rate and body temperature. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
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| Notes [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Change From Baseline in Z-Score of Body Weight at EOT [5] | ||||||||||
End point description |
A z-score is the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to 47.3-51.3 weeks)
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| Notes [5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||||||
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End point title |
Change From Baseline in Z-Score of Height at EOT [6] | ||||||||||
End point description |
A z-score is the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to 47.3-51.3 weeks)
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| 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: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||||||
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End point title |
Change From Baseline in Z-Score of Head Circumference at EOT [7] | ||||||||||
End point description |
A z-score is the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide. Number of subjects analysed is the number of participants with data available for analyses.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to 47.3-51.3 weeks)
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| Notes [7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||||||
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End point title |
Number of Participants With a Change in Stool Output Reported as an Adverse Event [8] | ||||||
End point description |
Urine and stool output was recorded and calculated in the output diary over a 48-hour period of PS and EN stability before every site visit and within 1 week of implementing a change in the PS prescription. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
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| Notes [8] - 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: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||
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End point title |
Change From Baseline in Z-Score of Weight-for-Length at EOT [9] | ||||||||||
End point description |
A z-score is the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide. Overall number analyzed is the number of participants with data available for analyses.
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End point type |
Primary
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End point timeframe |
Baseline, EOT (up to 47.3-51.3 weeks)
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| Notes [9] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||||||
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End point title |
Number of Participants With a Change in Urine Output Reported as an Adverse Event [10] | ||||||
End point description |
Urine and stool output was recorded and calculated in the output diary over a 48-hour period of parenteral support (PS) and enteral nutrition (EN) stability before every site visit and within 1 week of implementing a change in the PS prescription. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
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| Notes [10] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||
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End point title |
Number of Participants With any Laboratory Safety Finding Reported as an Adverse Event [11] | ||||||
End point description |
Laboratory safety parameters included biochemistry, hematology, and urinalysis. Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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End point type |
Primary
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End point timeframe |
From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
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| Notes [11] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive statistics were planned to be analysed for this endpoint. |
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| No statistical analyses for this end point | |||||||
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End point title |
Change From Baseline in PS Volume | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period. An end of treatment (EOT) was defined as the last determination of endpoint of the last cycle. Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. For Cycle 2, Week 24, n=2; Cycle 3, n=1.
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End point type |
Secondary
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End point timeframe |
Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2: Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Percent Change From Baseline in PS Volume | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Percent change from baseline in PS volume was calculated as follows; (PS volume at each point [Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT] - PS volume at baseline)/ PS volume at baseline *100 (percent). PS (parenteral nutrition or intravenous fluids) was to be considered for managing nutritional support in terms of volume and calories during the treatment period. An EOT was defined as the last determination of endpoint of the last cycle. Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. For Cycle 2, Week 24, n=2; Cycle 3, n=1.
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End point type |
Secondary
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End point timeframe |
Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Number of Participants who Demonstrate at least 20 Percent (%) Reduction From Baseline in PS Volume | ||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period. An EOT was defined as the last determination of endpoint of the last cycle. Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. For Cycle 2, Week 24, n=2; Cycle 3, n=1.
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End point type |
Secondary
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End point timeframe |
Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Number of Participants who Achieved Enteral Autonomy | ||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Achieving enteral autonomy is defined as complete weaning off PS. PS (parenteral nutrition or intravenous fluids) was to be considered for managing nutritional support in terms of volume and calories during the treatment period. Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. For Cycle 2, Week 24, n=2; Cycle 3, n=1.
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End point type |
Secondary
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End point timeframe |
Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Change from Baseline in Number of Days per Week of PS Usage at EOT | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period. Full Analysis Set included all enrolled participants, who were not screen failures, regardless of whether participants took any dose of teduglutide in the study. For Cycle 2, Week 24, n=2; Cycle 3, n=1.
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End point type |
Secondary
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End point timeframe |
Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT, and overall EOT (for 47.3-51.3 weeks) [cycle length=28 weeks]
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
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Adverse event reporting additional description |
Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
26.0
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Reporting groups
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Reporting group title |
Teduglutide
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Reporting group description |
Participants received teduglutide 0.05 milligram per kilogram (mg/kg) (0.025 mg/kg for participants with moderate or greater renal impairment) SC injection once daily for 24 weeks followed by no treatment period for 4 weeks or a maximum of 3 cycles (cycle length=24 weeks. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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01 Dec 2021 |
The following changes were implemented based on Amendment 1: 1. Changed the screening period. 2. Added the expected maximum duration of treatment (approximately 18 months). 3. Added a description to the estimated glomerular filtration rate criteria. 4. Amended the errors related to the change of screening period for another treatment cycle. 5. Removed “enteral glutamine” from exclusion criteria with considering the clinical settings in Japan. 6. Added a description to allow the participants who develop renal impairment during the study to continue the dosing. 7. Added a description to avoid the situation that teduglutide is administered twice a day with more than 12
hours separation. 8. Error modifications and description adjustments where applicable. |
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19 May 2022 |
The following changes were implemented based on Amendment 2: 1. Added a study procedure “evaluation of escape criteria” at Week 24. 2. Added the maximum duration of treatment. 3. Corrected a criterion of participants’ body weight, who develop renal impairment to continue the dosing during the study for consistency with the inclusion criterion 4. 4. Added a description to clarify that estimated glomerular filtration rates are calculated with the quintic equation. 5. Corrected typographical errors. |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
