UX007G-CL201 For Participants with Glut 1 DS
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Study to Assess the Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome
IRAS ID
172328
Contact name
Robert McFarland
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical Inc.
Eudract number
2013-003771-35
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
The purpose of this research study is to assess if an experimental drug called UX007 (triheptanoin) is an effective and safe treatment for participants aged 1 to 35 years 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 52 weeks. The study consists of a 6 week baseline period whereby participants will be required to record the frequency of their seizures. Suitable participants will then enter the 8 week treatment phase (participants will receive either placebo or UX007) which includes a 2 week titration period, followed by an open-label extension phase (all participants will receive UX007) for 44 weeks.
The 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
15/NE/0041
Date of REC Opinion
19 Mar 2015
REC opinion
Further Information Favourable Opinion