A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Clinical Trial

Official title:
An Open-label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Status Completed

Clinical Trial Summary

The primary objective of the study was to evaluate the impact of UX007 on acute clinical pathophysiology associated with LC-FAOD following 24 weeks of treatment.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Carnitine Palmitoyltransferase (CPT II) Deficiency
Disease
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
Trifunctional Protein (TFP) Deficiency
Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency

Clinical Trial Details

NCT number	NCT01886378
Study type	Interventional
Source	Ultragenyx Pharmaceutical Inc
Contact
Status	Completed
Phase	Phase 2
Start date	February 6, 2014
Completion date	August 25, 2016

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT04632953` - Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
Terminated	`NCT04812106` - Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
Completed	`NCT03768817` - Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
Recruiting	`NCT05933200` - A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD	Phase 3