Back to resultsStudy to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects With PN-dependent Short Bowel Syndrome (SBS)
Primary Purpose
Short Bowel Syndrome
Status
Completed
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
Teduglutide
Sponsored by
About this trial
This is an interventional treatment trial for Short Bowel Syndrome
Eligibility Criteria
This study has 3 stages, consisting of an optimization/stabilization period (Stage 1), a 24-week treatment period in which all patients will receive teduglutide (Stage 2), and a long-term extension (Stage 3).
During the long-term extension (Stage 3), patients will continue to receive teduglutide for up to an additional 24 months or until teduglutide is commercially available, whichever comes earlier.
Sites / Locations
- Tohoku University Hospital
- Yokohama Municipal Citizen's Hospital
- Hospital of Hyogo College of Medicine
- Osaka University Hospital, Department of Gastroenterological Surgery
- Osaka University Hospital, Department of Pediatric Surgery
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Arm 1
Arm Description
0.05 mg/kg/day administered by subcutaneous injection over a 24-week period.
Outcomes
Primary Outcome Measures
Stage 2: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
Absolute change from baseline in weekly PS volume at Week 24 was reported.
Stage 2: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
Percent change from baseline in weekly PS volume at Week 24 was reported.
Stage 2: Percentage of Participants Who Demonstrate Response to Teduglutide at End of Stage 2
Response was defined as the achievement of at least a 20% reduction from baseline (Visit 2) in weekly PS volume at Week 20 and again at Week 24.
Stage 2: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Week 24
Absolute change from baseline in number of days per Week of PS usage at Week 24 was reported.
Stage 2: Absolute Change From Baseline in Plasma Citrulline Levels at Week 24
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at Week 24 was reported.
Stage 2: Percent Change From Baseline in Plasma Citrulline Levels at Week 24
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change from baseline in plasma citrulline at Week 24 was reported.
Stage 2: Number of Participants Who Achieved Enteral Autonomy at Week 24
Enteral autonomy was defined as no prescribed PS and no use of PS recorded in the participant diary at the end of stage 2. Number of participants who achieved enteral autonomy at Week 24 was reported.
Stage 3: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24
Absolute change from baseline in weekly PS volume at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 3: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24
Percent change from baseline in weekly PS volume at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 3: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Extension Month 24
Absolute change from baseline in number of days per Week of PS usage at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 3: Absolute Change From Baseline in Plasma Citrulline Levels at Extension Month 24
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 3: Percent Change From Baseline in Plasma Citrulline Levels at Extension Month 24
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change from baseline in plasma citrulline at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Stage 4: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment
Absolute change from baseline in weekly PS volume at end of treatment was reported.
Stage 4: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment
Percent change from baseline in weekly PS volume at end of treatment was reported.
Stage 4: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at End of Treatment
Absolute change from baseline in number of days per week of PS usage at end of treatment was reported.
Stage 4: Absolute Change From Baseline in Plasma Citrulline Levels at End of Treatment
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at end of treatment was reported.
Stage 4: Percent Change From Baseline in Plasma Citrulline Levels at End of Treatment
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change in plasma citrulline from baseline at end of treatment was reported.
Secondary Outcome Measures
Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC0-Inf) of Teduglutide in Plasma
Area under the concentration-time curve from time zero to infinity of teduglutide in plasma were evaluated.
Area Under the Concentration-Time Curve From Time Zero to the Last Time Point (AUC0-t) of Teduglutide in Plasma
Area under the concentration-time curve from time zero to the last time point of teduglutide in plasma were evaluated.
Maximum Concentration (Cmax) of Teduglutide in Plasma
Maximum concentration of teduglutide in plasma were evaluated.
Time to Reach Maximum Observed Drug Concentration (Tmax) of Teduglutide in Plasma
Time to reach maximum observed drug concentration of teduglutide in plasma was evaluated.
Terminal Half-Life (t1/2) of Teduglutide in Plasma
Terminal half-life of teduglutide in plasma were evaluated.
Apparent Clearance (CL/F) of Teduglutide in Plasma
Apparent clearance of teduglutide in plasma were evaluated.
Apparent Volume of Distribution (Vz/F) of Teduglutide in Plasma
Apparent volume of distribution of teduglutide in plasma were evaluated.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02340819
Brief Title
Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects With PN-dependent Short Bowel Syndrome (SBS)
Official Title
A 4-Stage, Open-label, Multicenter Study Including Long-term Extension to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects With PN-dependent Short Bowel Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
December 18, 2014 (Actual)
Primary Completion Date
November 5, 2018 (Actual)
Study Completion Date
November 5, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of an investigational treatment (teduglutide) in Japanese patients with PN-dependent SBS. This study will also look at how teduglutide moves through the body (pharmacokinetics).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Short Bowel Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Arm 1
Arm Type
Experimental
Arm Description
0.05 mg/kg/day administered by subcutaneous injection over a 24-week period.
Intervention Type
Drug
Intervention Name(s)
Teduglutide
Intervention Description
0.05 mg/kg/day administered by subcutaneous injection over a 24-week period.
Primary Outcome Measure Information:
Title
Stage 2: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
Description
Absolute change from baseline in weekly PS volume at Week 24 was reported.
Time Frame
Baseline (stage 2), Week 24
Title
Stage 2: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
Description
Percent change from baseline in weekly PS volume at Week 24 was reported.
Time Frame
Baseline (stage 2), Week 24
Title
Stage 2: Percentage of Participants Who Demonstrate Response to Teduglutide at End of Stage 2
Description
Response was defined as the achievement of at least a 20% reduction from baseline (Visit 2) in weekly PS volume at Week 20 and again at Week 24.
Time Frame
End of Stage 2 (up to Week 24)
Title
Stage 2: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Week 24
Description
Absolute change from baseline in number of days per Week of PS usage at Week 24 was reported.
Time Frame
Baseline (stage 2), Week 24
Title
Stage 2: Absolute Change From Baseline in Plasma Citrulline Levels at Week 24
Description
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at Week 24 was reported.
Time Frame
Baseline (stage 2), Week 24
Title
Stage 2: Percent Change From Baseline in Plasma Citrulline Levels at Week 24
Description
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change from baseline in plasma citrulline at Week 24 was reported.
Time Frame
Baseline (stage 2), Week 24
Title
Stage 2: Number of Participants Who Achieved Enteral Autonomy at Week 24
Description
Enteral autonomy was defined as no prescribed PS and no use of PS recorded in the participant diary at the end of stage 2. Number of participants who achieved enteral autonomy at Week 24 was reported.
Time Frame
Week 24
Title
Stage 3: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24
Description
Absolute change from baseline in weekly PS volume at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Time Frame
Baseline (stage 3), Extension Month 24
Title
Stage 3: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24
Description
Percent change from baseline in weekly PS volume at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Time Frame
Baseline (stage 3), Extension Month 24
Title
Stage 3: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Extension Month 24
Description
Absolute change from baseline in number of days per Week of PS usage at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Time Frame
Baseline (stage 3), Extension Month 24
Title
Stage 3: Absolute Change From Baseline in Plasma Citrulline Levels at Extension Month 24
Description
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Time Frame
Baseline (stage 3), Extension Month 24
Title
Stage 3: Percent Change From Baseline in Plasma Citrulline Levels at Extension Month 24
Description
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change from baseline in plasma citrulline at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Time Frame
Baseline (stage 3), Extension Month 24
Title
Stage 4: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment
Description
Absolute change from baseline in weekly PS volume at end of treatment was reported.
Time Frame
Baseline (stage 4), End of Treatment (up to 47 months)
Title
Stage 4: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment
Description
Percent change from baseline in weekly PS volume at end of treatment was reported.
Time Frame
Baseline (stage 4), End of Treatment (up to 47 months)
Title
Stage 4: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at End of Treatment
Description
Absolute change from baseline in number of days per week of PS usage at end of treatment was reported.
Time Frame
Baseline (stage 4), End of Treatment (up to 47 months)
Title
Stage 4: Absolute Change From Baseline in Plasma Citrulline Levels at End of Treatment
Description
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at end of treatment was reported.
Time Frame
Baseline (stage 4), End of Treatment (up to 47 months)
Title
Stage 4: Percent Change From Baseline in Plasma Citrulline Levels at End of Treatment
Description
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change in plasma citrulline from baseline at end of treatment was reported.
Time Frame
Baseline (stage 4), End of Treatment (up to 47 months)
Secondary Outcome Measure Information:
Title
Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC0-Inf) of Teduglutide in Plasma
Description
Area under the concentration-time curve from time zero to infinity of teduglutide in plasma were evaluated.
Time Frame
Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Title
Area Under the Concentration-Time Curve From Time Zero to the Last Time Point (AUC0-t) of Teduglutide in Plasma
Description
Area under the concentration-time curve from time zero to the last time point of teduglutide in plasma were evaluated.
Time Frame
Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Title
Maximum Concentration (Cmax) of Teduglutide in Plasma
Description
Maximum concentration of teduglutide in plasma were evaluated.
Time Frame
Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Title
Time to Reach Maximum Observed Drug Concentration (Tmax) of Teduglutide in Plasma
Description
Time to reach maximum observed drug concentration of teduglutide in plasma was evaluated.
Time Frame
Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Title
Terminal Half-Life (t1/2) of Teduglutide in Plasma
Description
Terminal half-life of teduglutide in plasma were evaluated.
Time Frame
Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Title
Apparent Clearance (CL/F) of Teduglutide in Plasma
Description
Apparent clearance of teduglutide in plasma were evaluated.
Time Frame
Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Title
Apparent Volume of Distribution (Vz/F) of Teduglutide in Plasma
Description
Apparent volume of distribution of teduglutide in plasma were evaluated.
Time Frame
Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
This study has 3 stages, consisting of an optimization/stabilization period (Stage 1), a 24-week treatment period in which all patients will receive teduglutide (Stage 2), and a long-term extension (Stage 3).
During the long-term extension (Stage 3), patients will continue to receive teduglutide for up to an additional 24 months or until teduglutide is commercially available, whichever comes earlier.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Tohoku University Hospital
City
Sendai
State/Province
Aoba-ku
ZIP/Postal Code
980-8574
Country
Japan
Facility Name
Yokohama Municipal Citizen's Hospital
City
Yokohama
State/Province
Hodogaya-ku
ZIP/Postal Code
240-8555
Country
Japan
Facility Name
Hospital of Hyogo College of Medicine
City
Hyogo
State/Province
Nishinomiya
ZIP/Postal Code
663-8501
Country
Japan
Facility Name
Osaka University Hospital, Department of Gastroenterological Surgery
City
Osaka
State/Province
Suita
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Osaka University Hospital, Department of Pediatric Surgery
City
Osaka
State/Province
Suita
ZIP/Postal Code
565-0871
Country
Japan
12. IPD Sharing Statement
Plan to Share IPD
No
IPD Sharing Plan Description
De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites, …)
Learn more about this trial
Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects With PN-dependent Short Bowel Syndrome (SBS)
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