Teduglutide in Pediatric Subjects with Short Bowel Syndrome
Research type
Research Study
Full title
A 24-week Double-blind, Safety, Efficacy, and Pharmacodynamic Study Investigating Two Doses of Teduglutide in Pediatric Subjects Through 17 Years of Age with Short Bowel Syndrome who are Dependent on Parenteral Support
IRAS ID
206684
Contact name
Susan Hill
Contact email
Sponsor organisation
NPS Pharmaceuticals, Inc.
Eudract number
2015-002252-27
Duration of Study in the UK
0 years, 9 months, 19 days
Research summary
Short Bowel Syndrome (SBS) is a life-long condition caused by removal of a large part of the intestine that often leads to diarrhoea and poor growth. Patients with SBS need special fluids and nutrients in a bag, known as parenteral nutrition (PN/IV), given through the veins.
Although PN/IV can provide this nutritional support for patients with compromised fluid and nutritional status, it is also associated with serious complications, such as infections and liver damage.
Treatment of both children and adult patients is focused on achieving adequate intestinal absorption to allow for minimization or discontinuation of PN/IV support. Because children are at higher risk for intestinal failure-associated liver disease, reducing PN support is far more urgent in this population.
This phase III study proposes to investigate the safe and appropriate use of teduglutide, an experimental drug, in children and young adults through 17 years of age, for up to 24 weeks of administration for the purpose of providing longer-term data in regard to safety and the potential to explore further efficacy including PN reduction and the ability to completely wean off parenteral support.
This study will include 2 treatment arms: a teduglutide treatment arm and a standard of care (SOC) treatment arm. Approximately 26 subjects who have not had teduglutide before (teduglutide-naïve) will be enrolled into the teduglutide treatment arm and at least 8 subjects who have not had teduglutide before will be enrolled into the SOC treatment arm. Enrollment will proceed at approximately 28 investigational sites worldwide.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
16/SC/0308
Date of REC Opinion
19 Sep 2016
REC opinion
Further Information Favourable Opinion