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Open-label Long-Term Safety and Efficacy Extension Study of UX007 (triheptanoin) in LC-FAOD
2016-000322-19Phase 2Unknown
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Mar 2016
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of triheptanoin in long-chain fatty acid oxidation disorder (LC-FAOD), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Annualized LC-FAOD major events rate. Status: unknown.
02
Drugs
triheptanoin
03
Conditions
long-chain fatty acid oxidation disorder (LC-FAOD)
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