UX007G-CL202 extension study for Participants with Glut1 DS
Research type
Research Study
Full title
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome
IRAS ID
209692
Contact name
Robert McFarland
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical Inc.
Eudract number
2015-000389-69
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
Unique Product Identifer, EMA/190573
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
This extension study is linked to a previous study (IRAS 172328). The purpose of this research study is to allow continued treatment with the experimental drug called UX007(triheptanoin)and obtain further information on the effectiveness and safety of the UX007 used in children and adults with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS). UX007 is a liquid that will be mixed with food (or formula, as appropriate) and administered orally (PO) or by gastronomy tube at least four times per day (breakfast, lunch, dinner, and before bed) for a total of 3 years (36 months). The extension study consists of a baseline visits during which participants suitability will be assessed. Once enrolled participants will be treated with UX007 for up to three (3) years. Participants will have 3 monthly study visits in the first year, then every 6 monthly visit in year two (2) and three (3).
The extension study will enrol approximately 40 participants with Glut1 DS who are currently not on, or not fully compliant with a prescribed diet plan (ketogenic diet or other high fat diet). Enrolled participants are otherwise able to maintain standard of care treatment with up to 3 AEDs (antiepileptic drugs) throughout the duration of the study.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
16/NE/0259
Date of REC Opinion
27 Jan 2017
REC opinion
Further Information Favourable Opinion