Overview

Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Status:
Recruiting

Trial end date:
2025-04-01

Target enrollment:

Participant gender:

Summary

This is a medical research study to test a medication in adult patients with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD). The medication is triheptanoin, which is currently FDA approved for the treatment of Long-Chain Fatty Acid Oxidation Disorders. Previous research suggests that triheptanoin may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of triheptanoin in patients with MCADD.

Phase:

Phase 2

Details

Lead Sponsor:

Jerry Vockley, MD, PhD

Collaborator:

Ultragenyx Pharmaceutical Inc