SHP633-301
Research type
Research Study
Full title
A Randomized, Open-label, 24-Week Safety, Efficacy, and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age with Short Bowel Syndrome Who are Dependent on Parenteral Support Original PIP P/238/2010
IRAS ID
245151
Contact name
Susan Hill
Contact email
Sponsor organisation
Shire Human Genetic Therapies, Inc.
Eudract number
2017-003606-40
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
The purpose of this study is to assess the safety and efficacy of the study drug (Teduglutide) in Infants 4 to 12 months of age with Short Bowel Syndrome (SBS) who are dependent on Parenteral Support (PS).
There is no approved pharmacological therapy to improve intestinal adaptation in infants with SBS who are dependent on parenteral support. This study will evaluate whether teduglutide is safe and effective in this patient population.
The investigational drug, Teduglutide (TED) is already approved in the United States, Canada and the EU for the treatment of adults and children (over the age of 12 months) with SBS. TED is given as an injection under the skin once daily.This study is planned to be conducted in approximately 5 to 10 sites globally. In the UK, the aim is to open 4 sites in England. About 10 children are expected to take part in this study.
The study consists of a 2 to 4-week screening period, a 24-week treatment period, and a 4-week follow-up period. The maximum duration of participation for each potential participant is 32 weeks.
This is a randomised, open-label study, which means the doctor and the parents will know if the participant is assigned to the Study Drug Arm or to the Standard of care Arm.
Following the 4-week follow up period, the participant may enrol in an extension study that will provide additional TED treatment.
REC name
London - Chelsea Research Ethics Committee
REC reference
18/LO/0755
Date of REC Opinion
28 May 2018
REC opinion
Favourable Opinion