- 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
- UX007
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 7
- States / cities
- Washington D.C., District of Columbia • Tampa, Florida • Chicago, Illinois + 4 more
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- 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
- UX007
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 94 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2020
- U.S. locations
- 9
- States / cities
- San Francisco, California • Washington D.C., District of Columbia • Tampa, Florida + 6 more