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

Conditions

• Medium-chain Acyl-CoA Dehydrogenase Deficiency

Eligibility Criteria

Sex

ALL

Age

4 Years - N/A

Healthy Volunteers

Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date

Jul 31, 2026

Status verified

Mar 2026

Primary completion

Dec 31, 2027

Completion

Apr 30, 2028

Study Design

Enrollment

24 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: 1.0 gm/kg/day triheptanoin
  Up to 24 subjects (8: 4-9 years of age, 8: 10-15 years of age; 8: 16 years of age or older) will take 1.0 gm/kg/day divided into 3 or 4 doses

Primary Outcome Measure

Number of participants with treatment related adverse events as assessed by CTCAE v5.0 [ Time Frame: 8 weeks ]

Central Contacts

• Elizabeth McCracken
  412-692-5662
  [email protected]

Locations (1)

Find similar trials in Pittsburgh, PA

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