Medium chain acyl CoA dehydrogenase deficiency: Clinical Trials

2 withdrawn, 1 recruiting, 1 suspended. 4 Phase 2.

By phase and status

Phase	Trials	Status
Phase 2	4	Withdrawn 2, Recruiting 1, Suspended 1

Follow Medium chain acyl CoA dehydrogenase deficiency: new trials by email Literature Review of Medium chain acyl CoA dehydrogenase deficiency trials Research Report on Medium chain acyl CoA dehydrogenase deficiency

Trials (filtered) newest first · largest first

All 4 trials

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) Triheptanoin - Medium-chain Acyl-CoA Dehydrogenase Deficiency	Phase 2	Withdrawn	Jerry Vockley, MD, PhD		2026-08-01	2026-07-02
Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) Triheptanoin - Medium-chain Acyl-CoA Dehydrogenase Deficiency	Phase 2	Withdrawn	Jerry Vockley, MD, PhD		2026-07-01	2026-07-02
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) Sodium phenylbutyrate - Medium-chain Acyl-CoA Dehydrogenase Deficiency	Phase 2	Recruiting	Jerry Vockley, MD, PhD	24	2025-06-30	2026-10-21
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) Sodium phenylbutyrate - Medium-chain Acyl-CoA Dehydrogenase Deficiency	Phase 2	Suspended	Jerry Vockley, MD, PhD	24	2024-04-01	2026-07-06

Interventions

4-phenylbutyric acid (2)  ·  triheptanoin (2)

Sponsors

Jerry Vockley, MD, PhD (4)

Countries

United States (4)

Top investigators

Gerard Vockley, MD, PhD (2)  ·  Gerard L Vockley, MD, PhD (1)  ·  Gerard Vockley (1)