Trials / Completed
CompletedNCT01886378
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
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
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 29 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 6 Months
- Healthy volunteers
- Not accepted
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.
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
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | UX007 |
Timeline
- Start date
- 2014-02-06
- Primary completion
- 2016-08-25
- Completion
- 2016-08-25
- First posted
- 2013-06-25
- Last updated
- 2021-02-11
- Results posted
- 2021-02-11
Locations
10 sites across 2 countries: United States, United Kingdom
Source: ClinicalTrials.gov record NCT01886378. Inclusion in this directory is not an endorsement.