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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

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

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