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RECRUITING
NCT07097311
PHASE2

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

Sponsor: Jerry Vockley, MD, PhD

View on ClinicalTrials.gov

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.

Official title: A Phase II, Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Key Details

Gender

All

Age Range

4 Years - Any

Study Type

INTERVENTIONAL

Enrollment

24

Start Date

2026-07

Completion Date

2028-04

Last Updated

2026-03-05

Healthy Volunteers

No

Interventions

DRUG

Triheptanoin

Open-label design with doses of triheptanoin up to 1.0 gm/kg/day. The dose will be titrated from Day 1-24.

Locations (1)

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States