Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency clinical trials at UCSF
1 in progress, 0 open to new patients
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies.
Sorry, accepting new patients by invitation only
The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in LC-FAOD subjects. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in LC-FAOD and evaluate the impact of UX007 on clinical events associated with LC-FAOD.
San Francisco, California and other locations