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Trial intelligence / Profile preview
Triheptanoin vs MCT in Pediatric LC-FAOD
2023-509809-76-00Phase 3Authorised
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- May 2024
- Modality
- Small Molecules
- Countries
- 6
01
Overview
Phase 3, randomised, double-blind trial of triheptanoin in long-chain fatty acid oxidation disorder (LC-FAOD), Carnitine palmitoyltransferase 1A deficiency (CPT1A), carnitine palmitoyltransferase 2 deficiency (CPT2) and 4 more (2 arms), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Annualized event rate of major clinical events (MCEs). Planned start May 2024; status: authorised.
02
Drugs
triheptanoin
03
Conditions
long-chain fatty acid oxidation disorder (LC-FAOD)Carnitine palmitoyltransferase 1A deficiency (CPT1A)carnitine palmitoyltransferase 2 deficiency (CPT2)carnitine-acylcarnitine translocase deficiencyvery long-chain acyl-CoA dehydrogenase deficiency
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