Clinical trial NCT02949362: Long-term Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed the TED-C13-003 Study
Official title: A Retrospective and Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed TED-C13-003
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Short Bowel Syndrome
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2016-10-31
Enrollment: 29
Sex: ALL
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 teduglutide treatment of children with short bowel syndrome (SBS) who completed the TED-C13-003 study over a long-term period. It will evaluate how these children fared after the TED-C13-003 study ended. This study will also offer teduglutide treatment to eligible subjects, regardless of treatment received in TED-C13-003.


Eligibility criteria: Inclusion Criteria:

1. Subject provides written informed consent (subject, parent or legal guardian and, as appropriate, subject informed assent) to participate in the study before completing any study-related procedures.
2. Subject completed the TED-C13-003 study (including subjects in the standard of care treatment arm).
3. Subject understands and is willing and able to fully adhere to study requirements as defined in this protocol.

Exclusion Criteria:

There are no exclusion criteria for this study.

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs) in Retrospective Observation Period
timeFrame: From end of the core study (TED-C13-003 [NCT01952080]) up to the beginning of the prospective period (up to Week 168)
measure: Change From Baseline in Height for Age Z-score up to Week 168 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168)
measure: Change From Baseline in Body Weight for Age Z-score up to Week 168 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168)
measure: Change From Baseline in Body Mass Index (BMI) for Age Z-score up to Week 168 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168)
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period
timeFrame: From the beginning of the prospective study period to End of Study (EOS) (up to Week 144)
measure: Change From Baseline in Height for Age Z-score up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
measure: Change From Baseline in Body Weight for Age Z-score up to Cycle 6 Week 24 During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 24
measure: Change From Baseline in BMI for Age Z-score up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
measure: Average Total 48-Hour Urine Output up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
measure: Average Total 48-Hour Urine Output up to Week 108 During the End of Non-Teduglutide Treatment (NTT) Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period) up to Week 108
measure: Average Number of Stools Per Day up to Cycle 6 Week 24 During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 24
measure: Average Number of Stools Per Day up to Week 120 During the End of NTT Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period) up to Week 120
measure: Number of Participants With Positive Specific Antibodies at EOS During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: From the beginning of the prospective study period, EOS (up to Week 156)
Secondary outcomes:
measure: Number of Participants Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: At Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
measure: Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
measure: Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
measure: Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
measure: Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
measure: Change From Baseline in Number of Hours Per Day of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to Week 156
measure: Percent Change From Baseline in Number of Hours Per Day of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to Week 156
measure: Change From Baseline in Number of Days Per Week of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
measure: Percent Change From Baseline in Number of Days Per Week of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
timeFrame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
measure: Number of Participants Who Achieved At Least 20, 50 and 75% Reduction in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: At EOT of each Cycles 1 to 6 (up to Week 140) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 20 weeks)
measure: Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
measure: Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
measure: Number of Participants Who Achieved 100% Reduction in Complete Weaning of PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: At EOT of each Cycles 1 to 6 (up to Week 144) (length of each Cycle 1 to 6 = 24 weeks)
measure: Change From Baseline in Number of Hours Per Day of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Number of Hours Per Day of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
measure: Change From Baseline in Number of Days Per Week of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
measure: Percent Change From Baseline in Number of Days Per Week of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Other outcomes:
measure: Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Score at Week 12 and 24 of Each Treatment Cycle During the Teduglutide Treatment Periods of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6
measure: Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Score at End of Last NTT Period During the NTT Periods in Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), End of Last NTT period (up to Week 120)
measure: Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Family Impact Module Total Score at Week 12 and 24 of Each Treatment Cycle During the Teduglutide Treatment Periods of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6
measure: Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Family Impact Module Total Score at End of Last NTT Period During the NTT Periods in Prospective Study Period
timeFrame: Baseline (from the beginning of to the end the prospective study period), End of Last NTT period (up to Week 120)
measure: Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Gastrointestinal Symptoms Module at Week 12 and 24 of Each Treatment Cycle During the Teduglutide Treatment Periods of Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6
measure: Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Gastrointestinal Symptoms Module at End of Last NTT Period During the NTT Periods in Prospective Study Period
timeFrame: Baseline (from the beginning of the prospective study period), End of Last NTT period (up to Week 120)
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: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Kansas City
country: United States
facility: Children's Mercy Hospitals and Clinics
state: Missouri
city: Bronx
country: United States
facility: Children's Hospital at Montefiore
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
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: London
country: United Kingdom
facility: Great Ormond Children's Hospital
Study officials:
affiliation: Shire
name: Shire Study Physician
role: PRINCIPAL_INVESTIGATOR
References: