Teduglutide is approved for treatment of adults with short bowel syndrome (SBS). The purpose of this study is to evaluate the safety and efficacy of teduglutide in children up to the age of 17 with SBS who are dependent on parenteral support. Subjects may choose whether to receive the study drug or to participate in a standard-of-care arm. All participants who complete the study may be eligible to receive the study drug in a long-term extension study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
59
0.05 mg/kg
0.025 mg/kg
Observational cohort for the 24-week treatment period and 4 week follow-up.
Number of Participants Who Achieved at Least a 20 Percent (%) Reduction in Weight-Normalized Average Daily Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24
Reduction in weight-normalized PN/IV volume was performed using both participant diary and investigator prescribed data. Number of participants who achieved at least a 20% reduction in weight-normalized PN/IV volume between the baseline and week 24/EOT visit were reported.
Time frame: Baseline through Week 24
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs that started or worsened on or after the date of first dose for treatment groups and those that started or worsened on or after the baseline visit for standard of care group.
Time frame: From start of study treatment up to 28 weeks
Number of Participants Who Were Completely Weaned Off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
A participant was considered to have achieved independence from PN/IV support (completely weaned off PN/IV) if the investigator prescribed no PN/IV at EOT and there was no use of PN/IV recorded in the participant diary during the week prior to EOT.
Time frame: Week 24
Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24
Change in PN/IV volume was reported based on the participant diary and the investigator prescribed data.
Time frame: Baseline, Week 24
Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 24
Change in PN/IV caloric intake was reported based on the participant diary and the investigator prescribed data.
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Children's Hospital Los Angeles - RHU
Los Angeles, California, United States
UCLA Dept. of Medicine
Los Angeles, California, United States
UCSF Benioff Children's Hospital
San Francisco, California, United States
Georgetown Children's Research Network
Washington D.C., District of Columbia, United States
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
The Nebraska Medical Center
Omaha, Nebraska, United States
Children's Hospital GI Nutrition
New York, New York, United States
Montefiore Medical Center Child Spc
The Bronx, New York, United States
...and 17 more locations
Time frame: Baseline, Week 24
Change From Baseline in Plasma Citrulline Levels at Week 24
Plasma citrulline level was reported.
Time frame: Baseline, Week 24
Change From Baseline in Enteral Nutrition Volume at Week 24
Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition volume was reported.
Time frame: Baseline, Week 24
Change From Baseline in Enteral Nutrition Caloric Intake at Week 24
Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition caloric intake was reported.
Time frame: Baseline, Week 24
Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 28
Change in PN/IV volume was reported.
Time frame: Week 24, Week 28
Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 28
Change in PN/IV caloric intake was reported.
Time frame: Week 24, Week 28
Change From Week 24 in Plasma Citrulline Levels at Week 28
Change in plasma citrulline level was reported.
Time frame: Week 24, Week 28
Change From Week 24 in Enteral Nutrition Volume at Week 28
Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition volume was reported.
Time frame: Week 24, Week 28
Change From Week 24 in Enteral Nutrition Caloric Intake at Week 28
Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition caloric intake was reported.
Time frame: Week 24, Week 28
Change From Baseline in Body Weight Z-score at Week 28
Body weight z-score is a measure of relative weight adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.
Time frame: Baseline, Week 28
Change From Baseline in Body Height Z-score at Week 28
Body height z-score is a measure of relative height adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.
Time frame: Baseline, Week 28
Change From Baseline in Head Circumference Z-score at Week 28
Head circumference z-score is a measure of relative head circumference adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean. Head circumference was collected only for participants of less than or equal to (\<=) 36 months of age at the time of measurement.
Time frame: Baseline, Week 28
Change From Baseline in Body Mass Index (BMI) Z-score at Week 28
BMI z-score is a measure of relative BMI adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.
Time frame: Baseline, Week 28
Change From Baseline in Participants' Stool Consistency at Week 28
Stool consistency was assessed by typical stool form based on Bristol Stool Form Scale: 1 - Separate hard lumps, hard to pass, 2 - Sausage-shaped, but lumpy, 3 - Like a sausage but with cracks on the surface, 4 - Like a sausage or snake, smooth and soft, 5 - Soft blobs with clear-cut edges, 6 - Fluffy pieces with ragged edges, a mushy stool, 7 - Watery, no solid pieces, entirely liquid.
Time frame: Baseline, Week 28
Change From Baseline in Hours Per Day of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
The mean duration of the PN/IV infusions in hours, on the days when PN/IV was administered was reported.
Time frame: Baseline, Week 24
Change From Baseline in Days Per Week of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
The number of days per week of PN/IV infusions were reported.
Time frame: Baseline, Week 24