Trials / Completed
CompletedNCT02214160
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
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
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 94 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 6 Months
- Healthy volunteers
- Not accepted
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.
Conditions
- Carnitine 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
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | UX007 | Administered orally (PO) with food or by gastrostomy tube, at the target dose range of 25-35% of total calories. |
Timeline
- Start date
- 2014-12-09
- Primary completion
- 2020-12-03
- Completion
- 2020-12-03
- First posted
- 2014-08-12
- Last updated
- 2023-08-01
- Results posted
- 2021-12-08
Locations
11 sites across 2 countries: United States, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT02214160. Inclusion in this directory is not an endorsement.