Clinical trial NCT02954458: Long-term Safety and Efficacy Study of Teduglutide in Pediatric Participants With Short Bowel Syndrome (SBS)
Official title: A Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Pediatric Patients With Short Bowel Syndrome Who Completed TED-C14-006 or SHP633-301
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Short Bowel Syndrome
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2016-11-03
Enrollment: 61
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will follow participants who completed the TED-C14-006 study. The purpose of this study is to evaluate the long-term safety and efficacy of teduglutide in pediatric participants with Short Bowel Syndrome (SBS). This study will also offer teduglutide treatment to eligible participants, regardless of treatment received in TED-C14-006 or SHP633-301.


Eligibility criteria: Inclusion Criteria:

1. Participant provides written informed consent (participant, parent or legal guardian and, as appropriate, informed assent) to participate in the study before completing any study-related procedures.
2. Participant completed the TED-C14-006 or SHP633-301 studies (including participants in the standard of care treatment arms). Participants are considered to have completed SHP633-301 if they completed study assessments through week 24.
3. Participant understands and is willing and able to fully adhere to study requirements as defined in this protocol.

Exclusion Criteria:

1. There are no exclusion criteria for this study.

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: From start of study drug administration up to follow-up (up to 46 months)
measure: Change From Baseline in Average Total Urine Output at End of Treatment (EOT) of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Average Total Urine Output at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline in Average Number of Stools Per Day at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Average Number of Stools Per Day at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline in Average Total Daily Stool/Mixed Stool Diaper Weight at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Average Total Daily Stool/Mixed Stool Diaper Weight at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline in Average Total Ostomy Output at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Average Total Ostomy Output at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline in Average Bristol Stool Form Score at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Average Bristol Stool Form Score at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Number of Participants With Positive Specific Antibodies at End of Study (EOS)
timeFrame: At EOS (up to 46 months)
measure: Change From Baseline in Body Weight Z-score at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Body Weight Z-score at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline in Height Z-score at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Height Z-score at Last Visit During Non-Teduglutide Treatment
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline in Head Circumference Z-score at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Head Circumference Z-score at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline in Body Mass Index (BMI) Z-score at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 36) (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Body Mass Index (BMI) Z-score at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
Secondary outcomes:
measure: Number of Participants Who Achieved At Least 20 Percent (%), 50%, and 75% Reduction From Baseline in Diary Parenteral Support (PS) Volume at End of Treatment (EOT) of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Number of Participants Who Achieved At Least 20 Percent (%), 50%, and 75% Reduction From Baseline in Prescribed PS Volume at End of Treatment (EOT) of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Diary PS Caloric Intake at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Diary PS Caloric Intake at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Prescribed PS Caloric Intake at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Prescribed PS Caloric Intake at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Number of Participants Who Achieved 100% Reduction in Complete Weaning of PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Hours Per Day of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Hours Per Day of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Hours Per Day of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Hours Per Day of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Days Per Week of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Days Per Week of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline in Days Per Week of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Days Per Week of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
Other outcomes:
measure: Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Score at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline In PedsQL GCS Score at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline In PedsQL Total Family Impact Module Score at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline In PedsQL Total Family Impact Module Score at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
measure: Change From Baseline In PedsQL Gastrointestinal Symptoms Module at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, and 6 (Cycles 1, 2, 3, 4, and 5 = 28 weeks, and Cycle 6 = 16 weeks)
measure: Change From Baseline In PedsQL Gastrointestinal Symptoms Module at Last Visit During Non-Teduglutide Treatment (NT)
timeFrame: Baseline, Last visit in NT (up to Month 39)
Locations:
city: Los Angeles
country: United States
facility: Childrens Hospital Los Angeles - RHU
state: California
city: Los Angeles
country: United States
facility: Mattel Children's Hospital UCLA
state: California
city: San Francisco
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Washington
country: United States
facility: Georgetown Children's Research Network
state: District of Columbia
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Omaha
country: United States
facility: The Nebraska Medical Center
state: Nebraska
city: Bronx
country: United States
facility: Children's Hosp. at Montefiore
state: New York
city: New York
country: United States
facility: Columbia Unversity Medical Center
state: New York
city: Durham
country: United States
facility: Duke Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Madison
country: United States
facility: University of Wisconsin School of Medicine and Public Health
state: Wisconsin
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires Saint-Luc
state: Woluwe-Saint-Lambert
city: Edmonton
country: Canada
facility: Walter C. Mackenzie Health Science Centre
state: Alberta
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Helsinki
country: Finland
facility: Helsingin yliopistollinen keskussairaala
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesu
city: London
country: United Kingdom
facility: Great Ormond Children's Hosp
state: Greater London
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
state: West Midlands
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: