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

Shire11 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Short Bowel Syndrome

Interventions

TeduglutideDRUG

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

Eligibility

Sex: ALLMin age: 16 Years

Medical Language ↔ Plain English

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.

Locations (5)

Tohoku University Hospital

Sendai, Aoba-ku, Japan

Yokohama Municipal Citizen's Hospital

Yokohama, Hodogaya-ku, Japan

Hospital of Hyogo College of Medicine

Hyōgo, Nishinomiya, Japan

Osaka University Hospital, Department of Gastroenterological Surgery

Osaka, Suita, Japan

Outcomes

Primary Outcomes

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.

Time frame: Baseline (stage 2), Week 24

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.

Time frame: Baseline (stage 2), Week 24

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.

Time frame: End of Stage 2 (up to 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.

Time frame: Baseline (stage 2), Week 24

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.

Time frame: Baseline (stage 2), Week 24

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.

Time frame: Baseline (stage 2), Week 24

Stage 2: Number of Participants Who Achieved Enteral Autonomy at Week 24

Data from ClinicalTrials.gov

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Osaka University Hospital, Department of Pediatric Surgery

Osaka, Suita, Japan

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.

Time frame: Baseline (stage 3), Extension Month 24

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.

Time frame: Baseline (stage 3), Extension Month 24

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.

Time frame: Baseline (stage 3), Extension Month 24

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.

Time frame: Baseline (stage 3), Extension Month 24

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.

Time frame: Baseline (stage 3), Extension Month 24

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.

Time frame: Baseline (stage 4), End of Treatment (up to 47 months)

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.

Time frame: Baseline (stage 4), End of Treatment (up to 47 months)

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.

Time frame: Baseline (stage 4), End of Treatment (up to 47 months)

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.

Time frame: Baseline (stage 4), End of Treatment (up to 47 months)

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.

Time frame: Baseline (stage 4), End of Treatment (up to 47 months)

Secondary Outcomes

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.