Clinical trial NCT03268811: A Study in Japanese Children With Short Bowel Syndrome Who Completed SHP633-302
Official title: A Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Japanese Pediatric Subjects With Short Bowel Syndrome Who Completed SHP633-302
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Short Bowel Syndrome
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda
First posted: 2017-08-31
Enrollment: 9
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 clinical study is to evaluate the long-term safety and tolerability of teduglutide treatment in Japanese pediatric participants with short bowel syndrome (SBS) who completed Study SHP633-302 (NCT02980666).


Eligibility criteria: Inclusion Criteria:

* Informed consent by a parent or guardian prior to any study-related procedures.
* When applicable, informed assent (as deemed appropriate by the Institutional Review Board \[IRB\]) by the participant prior to any study-related procedures.
* Participant completed Study SHP633-302 (NCT02980666).
* Participant (and/or parent/guardian) 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 Treatment-emergent Adverse Events (TEAEs)
timeFrame: From Baseline up to follow-up (up to 50 months)
measure: Number of Participants With Clinically Significant Abnormalities in Vital Signs
timeFrame: From Baseline up to follow-up (up to 50 months)
measure: Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters
timeFrame: From Baseline up to follow-up (up to 50 months)
measure: Percent 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 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Percent 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 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Percent Change From Baseline in Average Stool/Mixed Stool Diaper Weight at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Percent 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 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Percent 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 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Number of Participants With Anti-drug Antibodies (ADAs) (Including Neutralizing Antibodies) at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Number of Participants With Clinically Significant Changes in Gastrointestinal (GI) Specific Testing
timeFrame: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Change From Baseline in Body Weight For Age Z-Score at EOT of Last Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 1 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 1 (up to 24 Weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 2 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 2 (up to 48 weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 3 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 3 (up to 72 weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 4 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 4 (up to 96 weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 5 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 5 (up to 120 weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 6 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 6 (up to 144 weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 7 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 7 (up to 168 weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 8 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 8 (up to 192 weeks)
measure: Change From Baseline in Height For Age Z-Score at EOT of Cycle 9 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 9 (up to 196 weeks)
measure: Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 1 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 1 (up to 24 weeks)
measure: Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 2 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 2 (up to 48 weeks)
measure: Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 3 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 3 (up to 72 weeks)
measure: Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 4 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 4 (up to 76 weeks)
measure: Change From Baseline in Body Mass Index (BMI) for Age Z-score at EOT of Cycle 1 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 1 (up to 24 weeks)
measure: Change From Baseline in BMI for Age Z-score at EOT of Cycle 2 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 2 (up to 48 weeks)
measure: Change From Baseline in BMI for Age Z-score at EOT of Cycle 3 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 3 (up to 72 weeks)
measure: Change From Baseline in BMI for Age Z-score at EOT of Cycle 4 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 4 (up to 96 weeks)
measure: Change From Baseline in BMI for Age Z-score at EOT of Cycle 5 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 5 (up to 120 weeks)
measure: Change From Baseline in BMI for Age Z-score at EOT of Cycle 6 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 6 (up to 144 weeks)
measure: Change From Baseline in BMI for Age Z-score at EOT of Cycle 7 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 7 (up to 168 weeks)
measure: Change From Baseline in BMI for Age Z-score at EOT of Cycle 8 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 8 (up to 192 weeks)
measure: Change From Baseline in BMI for Age Z-score at EOT of Cycle 9 During Teduglutide Treatment
timeFrame: Baseline, EOT of Cycle 9 (up to 196 weeks)
Secondary outcomes:
measure: Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Participants Diary Parenteral Support (PS) Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Investigator Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Change From Baseline in Participant Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Percent Change From Baseline in Participant Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Change From Baseline in Investigator Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Percent Change From Baseline in Investigator Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
measure: Number of Participants Who Achieved Enteral Autonomy PS Volume at EOT of Each Cycle During Teduglutide Treatment
timeFrame: Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 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, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 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, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Other outcomes:
Locations:
city: Akita-shi
country: Japan
facility: Akita University Hospital
state: Akita-ken
city: Fukuoka-shi
country: Japan
facility: Kyushu University Hospital
state: Fukuoka-ken
city: Tsukuba
country: Japan
facility: Tsukuba University Hospital
state: Ibaraki-Ken
city: Kagoshima
country: Japan
facility: Kagoshima University Hospital
state: Kagoshima-Ken
city: Sendai-shi
country: Japan
facility: Tohoku University Hospital
state: Miyagi-Ken
city: Shinagawa-ku
country: Japan
facility: Showa University Hospital
state: Tokyo-To
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: