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Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Sponsor: Jerry Vockley, MD, PhD
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
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