Clinical trial
An Open-label Long-Term Safety and Efficacy Extension Study in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies
NCT02214160 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- An Open-label Long-Term Safety and Efficacy Extension Study in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Phase
- Phase 2
- Overall status
- Completed
- Start date
- December 9, 2014
- Completion date
- December 3, 2020
- Enrollment
- 94 (actual)
- Sites
- 11
Conditions
As the registry lists them; a condition with its own page is linked.
carnitine palmitoyltransferase (cpt i or cpt ii) deficiency, carnitine-acylcarnitine translocase (cact) deficiency, long-chain 3-hydroxy-acyl-coa dehydrogenase (lchad) deficiency, trifunctional protein (tfp) deficiency, very long chain acyl-coa dehydrogenase (vlcad) deficiency