Could a liver drug help kids and adults with rare metabolic disorder?

NCT ID NCT06773026

Recruiting now Disease control Sponsor: Jerry Vockley, MD, PhD Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether sodium phenylbutyrate (ACER-001), a drug already approved for another condition, can help people with MCAD deficiency caused by a specific gene mutation. About 24 participants aged 4 and older will take the drug and be monitored for safety and how well it keeps their blood sugar stable. The goal is to see if this drug can reduce the risk of dangerous low blood sugar episodes.

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Conditions

The condition(s) this trial relates to.

medium chain acyl-CoA dehydrogenase deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

UPMC Children's Hospital of Pittsburgh
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact