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

NCT02340819 | Completed Phase 3 Results

• 1 other identifier: TED-C14-004
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 5

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).

Conditions

Short Bowel Syndrome

Outcome Measures

Primary Outcomes (17)

• 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

• 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

• 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)

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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)

• 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)

• 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)

• 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)

• 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 Outcomes (7)

• 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 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

• Maximum Concentration (Cmax) 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 (Tmax) of Teduglutide in Plasma

  Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose

• 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

Study Arms (1)

Arm 1

EXPERIMENTAL

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

Drug: Teduglutide

Interventions

Teduglutide DRUG

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

Arm 1

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Shire: lead

Study Sites (5)

Tohoku University Hospital

Sendai, Aoba-ku, 980-8574, Japan

Location

Yokohama Municipal Citizen's Hospital

Yokohama, Hodogaya-ku, 240-8555, Japan

Location

Hospital of Hyogo College of Medicine

Hyōgo, Nishinomiya, 663-8501, Japan

Location

Osaka University Hospital, Department of Gastroenterological Surgery

Osaka, Suita, 565-0871, Japan

Location

Osaka University Hospital, Department of Pediatric Surgery

Osaka, Suita, 565-0871, Japan

Location

MeSH Terms

Conditions

Short Bowel Syndrome

Interventions

teduglutide

Condition Hierarchy (Ancestors)

Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2014
• First Posted: January 19, 2015
• Study Start: December 18, 2014
• Primary Completion: November 5, 2018
• Study Completion: November 5, 2018
• Last Updated: June 9, 2021
• Results First Posted: November 20, 2019

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share

Locations

JP (5)