SHP633-303 Ext Study of Teduglutide in Paediatric Subjects with SBS
Research type
Research Study
Full title
A Retrospective and Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Pediatric Subjects with Short Bowel Syndrome Who Completed TED-C13-003
IRAS ID
219464
Contact name
Susan Hill
Contact email
Sponsor organisation
Shire Human Genetic Therapies, Inc.
Eudract number
2016-000863-17
Clinicaltrials.gov Identifier
058213, IND
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Short Bowel Syndrome (SBS) is a life-long condition caused by loss of most of the small intestine that often leads to diarrhoea and poor growth. Patients with SBS need special fluids and nutrients in a bag, known as parenteral support (PS), given through the veins (IV).
Although PS can provide 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 adults is focused on achieving adequate intestinal absorption to allow for minimisation or discontinuation of PS. Because children are at higher risk for intestinal failure-associated liver disease, reducing parenteral support is far more urgent in this population.
This phase III extension study proposes to continue to investigate the safe and appropriate use of teduglutide in children and young adults who completed TED-C13-003 study. Teduglutide is already approved in US and Canada to treat SBS in adults and in the EU to treat both adults and children.
This study is divided in 2 parts. In the first part, called retrospective part, information about patients from the time they completed TED-C13-003 study up to now, will be collected. This will include data such as weight, height, dates and doses of teduglutide and PS prescriptions and details of any medical events patients experienced.
The second part, called prospective part of the study, is done to test how safe and efficacious study medicine is when taken for a long period of time, and whether it will allow to lower the amount of PS patients are receiving. In part 1 patients will not take any study drug and will attend only one study visit. In part 2 patients will be attending study visits every 1-4 weeks during a study drug treatment cycle, and approx. every 12 weeks outside of these cycles over 3 years period. Patients will be required to have blood tests performed and possibly some extra investigations such as a colonoscopy or sigmoidoscopy. Patients will have to agree to join part 1 of the study before they can participate in part 2.
About 40 children, who were younger than 18 years of age at the time they started TED-C13-003 study, in the US and UK, are expected to take part in this study.REC name
London - City & East Research Ethics Committee
REC reference
17/LO/0121
Date of REC Opinion
28 Mar 2017
REC opinion
Further Information Favourable Opinion