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)

Official 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)

UX007-CL201 is an open-label Phase 2 study to assess the safety and clinical effects of UX007 in subjects with LC-FAOD. Following a 4 week run in period on current therapy, subjects will cross over to daily UX007 treatment for an initial 24 week treatment period, followed by an additional 54 week extension period. Approximately 30 subjects at least 6 months of age inclusive with severe LC-FAOD, specifically VLCAD, LCHAD, CPT 2, or TFP disorders, will be enrolled and treated with UX007.
NCT01886378
Burton, Barbara K., MD
Interventional
No

Contact Information:

Barbara K. Burton, MD

312.227.6124