Conditions: Long-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
Keywords: FAOD VLCAD CPT II CPT2 CPT 2 LCHAD UX007 Triheptanoin TFP C7
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 29
Healthy volunteers: no

Brief 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.

Detailed description:

Arms:
arm 1: UX007 dosing titrated to a target dose of 25-35% of total caloric intake or maximum tolerated dose. Participants are followed to evaluate the effects of UX007 over 24 weeks (Treatment Period), then continued treatment in the Extension Period for an additional 54 weeks for a total of 78 weeks of treatment.

Interventions: intervention 1: UX007
Intervention descriptions: intervention 1: None

Protocol document: