At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 29 enrolled
Drug / intervention
UX007drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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)
In Brief
A Phase 2 clinical trial evaluating UX007 for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) and 4 related conditions. Completed, enrolled 29 participants across 10 sites in 2 countries.
Detailed 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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsLong-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency
CountriesUnited Kingdom, United States
Collaborators--
Timeline
Phase 2CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedJun 2013
Enrollment StartFeb 2014
Primary CompletionAug 2016
TodayJul 2026
First PostedJun 25, 2013
Enrollment StartFeb 6, 2014
Primary CompletionAug 25, 2016
TodayJul 2, 2026
Enrollment to primary: 2.6 yearsPosted 13.0 years ago
Interventions
UX007drug