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

Trifunctional Protein (TFP) Deficiency Clinical Trial

Official 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

Status Completed

Clinical Trial Summary

The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. 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.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• 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

• NCT number: NCT02214160
• Study type: Interventional
• Source: Ultragenyx Pharmaceutical Inc
• Status: Completed
• Phase: Phase 2
• Start date: December 9, 2014
• Completion date: December 3, 2020