Overview

Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies.

Status:
Completed
Trial end date:
2020-10-19
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Ultragenyx Pharmaceutical Inc