Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects With PN-dependent Short Bowel Syndrome (SBS)
- Registration Number
- NCT02340819
- Lead Sponsor
- Shire
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 11
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Arm 1 Teduglutide 0.05 mg/kg/day administered by subcutaneous injection over a 24-week period.
- Primary Outcome Measures
Name Time Method Stage 2: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24 Baseline (stage 2), Week 24 Percent change from baseline in weekly PS volume at Week 24 was reported.
Stage 2: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Week 24 Baseline (stage 2), Week 24 Absolute change from baseline in number of days per Week of PS usage at Week 24 was reported.
Stage 2: Number of Participants Who Achieved Enteral Autonomy at Week 24 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 2: Percent Change From Baseline in Plasma Citrulline Levels at Week 24 Baseline (stage 2), 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 3: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24 Baseline (stage 3), 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 Baseline (stage 3), 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 2: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24 Baseline (stage 2), Week 24 Absolute 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 End of Stage 2 (up to Week 24) 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 Plasma Citrulline Levels at Week 24 Baseline (stage 2), 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 3: Absolute Change From Baseline in Plasma Citrulline Levels at Extension Month 24 Baseline (stage 3), 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 Baseline (stage 3), 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 3: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24 Baseline (stage 3), 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 4: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment Baseline (stage 4), End of Treatment (up to 47 months) 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 Baseline (stage 4), End of Treatment (up to 47 months) 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 Baseline (stage 4), End of Treatment (up to 47 months) 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 Baseline (stage 4), End of Treatment (up to 47 months) 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 Baseline (stage 4), End of Treatment (up to 47 months) 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
Name Time Method Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC0-Inf) of Teduglutide in Plasma Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose 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 Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose 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 Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose Maximum concentration of teduglutide in plasma were evaluated.
Time to Reach Maximum Observed Drug Concentration (Tmax) of Teduglutide in Plasma Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose Time to reach maximum observed drug concentration of teduglutide in plasma was evaluated.
Terminal Half-Life (t1/2) of Teduglutide in Plasma Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose Terminal half-life of teduglutide in plasma were evaluated.
Apparent Clearance (CL/F) of Teduglutide in Plasma Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose Apparent clearance of teduglutide in plasma were evaluated.
Apparent Volume of Distribution (Vz/F) of Teduglutide in Plasma Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose Apparent volume of distribution of teduglutide in plasma were evaluated.
Trial Locations
- Locations (5)
Hospital of Hyogo College of Medicine
🇯🇵Hyogo, Nishinomiya, Japan
Osaka University Hospital, Department of Pediatric Surgery
🇯🇵Osaka, Suita, Japan
Tohoku University Hospital
🇯🇵Sendai, Aoba-ku, Japan
Yokohama Municipal Citizen's Hospital
🇯🇵Yokohama, Hodogaya-ku, Japan
Osaka University Hospital, Department of Gastroenterological Surgery
🇯🇵Osaka, Suita, Japan