Phase 2 Study of UX007 in Subjects with LC-FAOD
Research type
Research Study
Full title
An Open-Label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
IRAS ID
145968
Contact name
Anupam Chakrapani
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical
Eudract number
2013-004830-14
Clinicaltrials.gov Identifier
Research summary
UX007-CL201 is an open-label phase 2 study to assess the safety and clinical effects of UX007 in subjects with long-chain fatty acid oxidation disorders (LC-FAOD). UX007 will be administered orally with food or by gastronomy tube at least 4 times per day for a 24 week treatment period, with the option to continue treatment for an additional 54 week extension period (78 week total treatment duration).
Approximately 30 subjects globally, who are between 6 months and 35 years of age inclusive, will be enrolled and treated with UX007. To qualify, subjects must have severe LC-FAOD, specifically VLCAD, LCHAD, or CPT2, and continue to exhibit clinical manifestations of LC-FAOD despite current therapy.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
14/WM/0022
Date of REC Opinion
26 Feb 2014
REC opinion
Further Information Favourable Opinion