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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