At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 94 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 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
In Brief
A Phase 2 clinical trial evaluating UX007 for Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency and 4 related conditions. Completed, enrolled 94 participants across 11 sites in 2 countries.
Detailed 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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsCarnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency, Carnitine-acylcarnitine Translocase (CACT) Deficiency
CountriesUnited Kingdom, United States
Collaborators--
Timeline
Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedAug 2014
Enrollment StartDec 2014
Primary CompletionDec 2020
TodayJul 2026
First PostedAug 12, 2014
Enrollment StartDec 9, 2014
Primary CompletionDec 3, 2020
TodayJul 2, 2026
Enrollment to primary: 6.0 yearsPosted 11.9 years ago
Interventions
UX007drug
Administered orally (PO) with food or by gastrostomy tube, at the target dose range of 25-35% of total calories.