Clinical Trial

Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Withdrawn Phase 2
View on ClinicalTrials.gov →
Why the trial stopped
funding was pulled
Summary
This is a medical research study to test a medication in patients 4 years of age and older 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 (FAODs). 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 patients with MCADD.
Protocol Amendment History 2 changes
critical Trial status changed: Recruiting → Withdrawn 2026-07-03
notable Enrollment reduced: 24 -> 0 participants 2026-07-03
Trial Details
NCT Number NCT07097311
Lead Sponsor Jerry Vockley, MD, PhD
Collaborators: Ultragenyx Pharmaceutical Inc
Conditions Medium-chain Acyl-CoA Dehydrogenase Deficiency
Start Date 2026-07
Primary Completion 2027-12 (estimated)
Study Completion 2028-04 (estimated)
Updated on ClinicalTrials.gov 2026-07-02