Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects With PN-dependent Short Bowel Syndrome (SBS)

Short Bowel Syndrome Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02340819
• Other study ID #: TED-C14-004
• Status: Completed
• Phase: Phase 3
• Start date: December 18, 2014
• Est. completion date: November 5, 2018

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: November 5, 2018
• Est. primary completion date: November 5, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Short Bowel Syndrome
• Syndrome

Intervention

Drug:
• Teduglutide
0.05 mg/kg/day administered by subcutaneous injection over a 24-week period.

Locations

Country	Name	City	State
Japan	Hospital of Hyogo College of Medicine	Hyogo	Nishinomiya
Japan	Osaka University Hospital, Department of Gastroenterological Surgery	Osaka	Suita
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

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline (stage 2), Week 24
Primary	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.	Baseline (stage 2), Week 24
Primary	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.	End of Stage 2 (up to Week 24)
Primary	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.	Baseline (stage 2), Week 24
Primary	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.	Baseline (stage 2), Week 24
Primary	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.	Baseline (stage 2), Week 24
Primary	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.	Week 24
Primary	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.	Baseline (stage 3), Extension Month 24
Primary	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.	Baseline (stage 3), Extension Month 24
Primary	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.	Baseline (stage 3), Extension Month 24
Primary	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.	Baseline (stage 3), Extension Month 24
Primary	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.	Baseline (stage 3), Extension Month 24
Primary	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.	Baseline (stage 4), End of Treatment (up to 47 months)
Primary	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.	Baseline (stage 4), End of Treatment (up to 47 months)
Primary	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.	Baseline (stage 4), End of Treatment (up to 47 months)
Primary	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.	Baseline (stage 4), End of Treatment (up to 47 months)
Primary	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.	Baseline (stage 4), End of Treatment (up to 47 months)
Secondary	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.	Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Secondary	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.	Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Secondary	Maximum Concentration (Cmax) of Teduglutide in Plasma	Maximum concentration of teduglutide in plasma were evaluated.	Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Secondary	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.	Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Secondary	Terminal Half-Life (t1/2) of Teduglutide in Plasma	Terminal half-life of teduglutide in plasma were evaluated.	Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Secondary	Apparent Clearance (CL/F) of Teduglutide in Plasma	Apparent clearance of teduglutide in plasma were evaluated.	Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Secondary	Apparent Volume of Distribution (Vz/F) of Teduglutide in Plasma	Apparent volume of distribution of teduglutide in plasma were evaluated.	Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose