DRUG
Teduglutide 0.05mg/kg
0.05 mg/kg
Status
Completed
Phase
Phase 3
Enrollment
59
Locations
27
Results
Posted
Publications
1
Study summary
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.
Interventions
DRUG
0.05 mg/kg
DRUG
0.025 mg/kg
OTHER
Observational cohort for the 24-week treatment period and 4 week follow-up.
Timeline
First posted
Feb 15, 2016
Study start
Jun 23, 2016
Primary completion
Aug 18, 2017
Study completion
Aug 18, 2017
Results posted
Oct 16, 2018
Registry updated
Jun 9, 2021
Outcomes
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
Time frame · Baseline through 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.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame · From start of study treatment up to 28 weeks
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.
Number of Participants Who Were Completely Weaned Off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
Time frame · 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.
Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24
Time frame · Baseline, Week 24
Change in PN/IV volume was reported based on the participant diary and the investigator prescribed data.
Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 24
Time frame · Baseline, Week 24
Change in PN/IV caloric intake was reported based on the participant diary and the investigator prescribed data.
Change From Baseline in Plasma Citrulline Levels at Week 24
Time frame · Baseline, Week 24
Plasma citrulline level was reported.
Change From Baseline in Enteral Nutrition Volume at Week 24
Time frame · Baseline, 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.
Change From Baseline in Enteral Nutrition Caloric Intake at Week 24
Time frame · Baseline, 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.
Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 28
Time frame · Week 24, Week 28
Change in PN/IV volume was reported.
Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 28
Time frame · Week 24, Week 28
Change in PN/IV caloric intake was reported.
Change From Week 24 in Plasma Citrulline Levels at Week 28
Time frame · Week 24, Week 28
Change in plasma citrulline level was reported.
Eligibility
Inclusion Criteria: 1. Informed consent by a parent or guardian or emancipated minor prior to any study-related procedures 2. When applicable, an informed assent by the subject (as deemed appropriate by the Ethics Committee/Institutional Review Board) prior to any study-related procedures 3. Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis) 4. Short bowel syndrome that requires PN/IV support that provides at least 30% of caloric and/or fluid/electrolyte needs prior to screening 5. Stable PN/IV support, defined as inability to significantly reduce PN/IV support, usually associated with minimal or no advance in enteral feeds (ie, 10% or less change in PN or advance in feeds) for at least 3 months prior to and during screening, as assessed by the investigator. 6. Sexually active female subjects of child-bearing potential (in the teduglutide treatment arm only) must use medically acceptable methods of birth control during and 4 weeks after the treatment period Exclusion Criteria: 1. Subjects who are not expected to be able to advance oral or tube feeding regimens 2. Serial transverse enteroplasty or any other bowel lengthening procedure performed within 3 months of screening 3. Known clinically significant untreated intestinal obstruction contributing to feeding intolerance and inability to reduce parenteral support 4. Unstable absorption due to cystic fibrosis or known DNA abnormalities 5. Severe, known dysmotility syndrome, such as pseudo-obstruction or persistent, severe, active gastroschisis-related dysmotility, that is the primary contributing factor to feeding intolerance and inability to reduce parenteral support, prior to screening. Dysmotility is defined as severe if it is expected to limit the advancement of enteral feeding. 6. Evidence of clinically significant obstruction on upper GI series done within 6 months prior to screening. 7. Major GI surgical intervention including significant intestinal resection within 3 months prior to the screening visit (insertion of feeding tube, anastomotic ulcer repair, minor intestinal resections ≤ 10 cm, or endoscopic procedure is allowed). 8. Unstable cardiac disease, congenital heart disease or cyanotic disease, with the exception of subjects who had undergone ventricular or atrial septal defect repair, and patent ductus arteriosus (PDA) ligation. 9. History of cancer or clinically significant lymphoproliferative disease, not including resected cutaneous basal or squamous cell carcinoma, or in situ non aggressive and surgically resected cancer. 10. Pregnant or lactating female subjects (in the teduglutide treatment arm only). 11. Participation in a clinical study using an experimental drug (other than glutamine or Omegaven) within 3 months or 5.5 half-lives of the experimental drug, whichever is longer, prior to screening, and for the duration of the study. 12. Previous use of teduglutide or native/synthetic glucagon-like peptide-2 (GLP-2) 13. Previous use of glucagon-like peptide-1 analog or human growth hormone within 3 months prior to screening 14. Previous use of octreotide, or dipeptidyl peptidase-4 (DPP-4) inhibitors within 3 months prior to screening 15. Subjects with active Crohn's disease who had been treated with biological therapy (eg, antitumor necrosis factor \[anti-TNF\]) within the 6 months prior to the screening visit 16. Subjects with inflammatory bowel disease (IBD) who require chronic systemic immunosuppressant therapy that had been introduced or changed during the 3 months prior to screening 17. More than 3 SBS-related or PN-related hospital admissions (eg, documented infection-related catheter sepsis, clots, bowel obstruction, severe water-electrolyte disturbances) within 3 months prior to the screening visit 18. Any major unscheduled hospital admission which affects parenteral support requirements within 1 month prior to or during screening, excluding uncomplicated treatment of bacteremia, central line replacement/repair, or issues of similar magnitude in an otherwise stable subject 19. Body weight \< 10 kg at the screening and baseline visits 20. Signs of active severe or unstable, clinically significant hepatic impairment during the screening period, as indicated by any of the following laboratory test results : 1. Total bilirubin (TBL) ≥ 2 x upper limit of normal (ULN) 2. Aspartate aminotransferase (AST) ≥ 7x ULN 3. Alanine aminotransferase (ALT) ≥ 7x ULN For subjects with Gilbert's disease: 4. Indirect (unconjugated) bilirubin ≥ 2x ULN 21. Signs of known continuous active or unstable, clinically significant renal dysfunction shown by results of an estimated glomerular filtration rate (eGFR) below 50 mL/min/1.73 m2. 22. Parent(s) and/or subjects who are not capable of understanding or not willing to adhere to the study visit schedule and other protocol requirements 23. Unstable, clinically significant active, untreated pancreatic or biliary disease 24. Any condition, disease, illness, or circumstance that in the investigator's opinion puts the subject at any undue risk, prevents completion of the study, or interferes with analysis of the study results.
Study locations
Belgium · Canada · Finland · Germany · Italy · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
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
Duke Medical Center
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic Pediatric Specialists
Cleveland, Ohio, United States
University of Pennsylvania Medical Center
Philadelphia, Pennsylvania, United States
Children's Medical Center Dallas
Dallas, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
University of Wisconsin School of Medicine and Public Health
Madison, Wisconsin, United States
Cliniques Universitaires Saint-Luc
Brussels, Belgium
Walter C. Mackenzie Health Science Center
Edmonton, Alberta, Canada
British Columbia Children's & Women's Hospital Center
Vancouver, British Columbia, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
Helsingin yliopistollinen keskussairaala
Helsinki, Finland
Universitaetsklinikum Tuebingen
Tübingen, Baden Wuertternberg, Germany
Publications
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