Clinical trial NCT02340819: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Short Bowel Syndrome
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2015-01-19
Enrollment: 11
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


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.

Primary outcomes:
measure: Stage 2: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
timeFrame: Baseline (stage 2), Week 24
measure: Stage 2: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Week 24
timeFrame: Baseline (stage 2), Week 24
measure: Stage 2: Percentage of Participants Who Demonstrate Response to Teduglutide at End of Stage 2
timeFrame: End of Stage 2 (up to Week 24)
measure: Stage 2: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Week 24
timeFrame: Baseline (stage 2), Week 24
measure: Stage 2: Absolute Change From Baseline in Plasma Citrulline Levels at Week 24
timeFrame: Baseline (stage 2), Week 24
measure: Stage 2: Percent Change From Baseline in Plasma Citrulline Levels at Week 24
timeFrame: Baseline (stage 2), Week 24
measure: Stage 2: Number of Participants Who Achieved Enteral Autonomy at Week 24
timeFrame: Week 24
measure: Stage 3: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24
timeFrame: Baseline (stage 3), Extension Month 24
measure: Stage 3: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at Extension Month 24
timeFrame: Baseline (stage 3), Extension Month 24
measure: Stage 3: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at Extension Month 24
timeFrame: Baseline (stage 3), Extension Month 24
measure: Stage 3: Absolute Change From Baseline in Plasma Citrulline Levels at Extension Month 24
timeFrame: Baseline (stage 3), Extension Month 24
measure: Stage 3: Percent Change From Baseline in Plasma Citrulline Levels at Extension Month 24
timeFrame: Baseline (stage 3), Extension Month 24
measure: Stage 4: Absolute Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment
timeFrame: Baseline (stage 4), End of Treatment (up to 47 months)
measure: Stage 4: Percent Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment
timeFrame: Baseline (stage 4), End of Treatment (up to 47 months)
measure: Stage 4: Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at End of Treatment
timeFrame: Baseline (stage 4), End of Treatment (up to 47 months)
measure: Stage 4: Absolute Change From Baseline in Plasma Citrulline Levels at End of Treatment
timeFrame: Baseline (stage 4), End of Treatment (up to 47 months)
measure: Stage 4: Percent Change From Baseline in Plasma Citrulline Levels at End of Treatment
timeFrame: Baseline (stage 4), End of Treatment (up to 47 months)
Secondary outcomes:
measure: Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC0-Inf) of Teduglutide in Plasma
timeFrame: Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
measure: Area Under the Concentration-Time Curve From Time Zero to the Last Time Point (AUC0-t) of Teduglutide in Plasma
timeFrame: Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
measure: Maximum Concentration (Cmax) of Teduglutide in Plasma
timeFrame: Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
measure: Time to Reach Maximum Observed Drug Concentration (Tmax) of Teduglutide in Plasma
timeFrame: Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
measure: Terminal Half-Life (t1/2) of Teduglutide in Plasma
timeFrame: Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
measure: Apparent Clearance (CL/F) of Teduglutide in Plasma
timeFrame: Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
measure: Apparent Volume of Distribution (Vz/F) of Teduglutide in Plasma
timeFrame: Pre-dose, 15, 30, 60 minutes, 2, 3, 4, 6, 8, 10 and 12 hours post-dose
Other outcomes:
Locations:
city: Sendai
country: Japan
facility: Tohoku University Hospital
state: Aoba-ku
city: Yokohama
country: Japan
facility: Yokohama Municipal Citizen's Hospital
state: Hodogaya-ku
city: Hyogo
country: Japan
facility: Hospital of Hyogo College of Medicine
state: Nishinomiya
city: Osaka
country: Japan
facility: Osaka University Hospital, Department of Gastroenterological Surgery
state: Suita
city: Osaka
country: Japan
facility: Osaka University Hospital, Department of Pediatric Surgery
state: Suita
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: