Last Updated: November 8, 2026

CLINICAL TRIALS PROFILE FOR OLPRUVA

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All Clinical Trials for OLPRUVA

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Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT06069375 ↗	Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)	Recruiting	Acer Therapeutics Inc.	Phase 2	2024-04-01	This is a medical research study to test a medication in patients 10 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD.
NCT06069375 ↗	Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)	Recruiting	Jerry Vockley, MD, PhD	Phase 2	2024-04-01	This is a medical research study to test a medication in patients 10 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD.
>Trial ID	>Title	>Status	>Sponsor	>Phase	>Start Date	>Summary

Clinical Trial Conditions for OLPRUVA

Condition Name

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Condition Name for OLPRUVA
Intervention	Trials
Medium-chain Acyl-CoA Dehydrogenase Deficiency	1
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Condition MeSH

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Condition MeSH for OLPRUVA
Intervention	Trials
Lipid Metabolism, Inborn Errors	1
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Clinical Trial Locations for OLPRUVA

Trials by Country

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Trials by Country for OLPRUVA
Location	Trials
United States	1
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Trials by US State

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Trials by US State for OLPRUVA
Location	Trials
Pennsylvania	1
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Clinical Trial Progress for OLPRUVA

Clinical Trial Phase

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Clinical Trial Phase for OLPRUVA
Clinical Trial Phase	Trials
Phase 2	1
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Clinical Trial Status

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Clinical Trial Status for OLPRUVA
Clinical Trial Phase	Trials
Recruiting	1
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Clinical Trial Sponsors for OLPRUVA

Sponsor Name

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Sponsor Name for OLPRUVA
Sponsor	Trials
Acer Therapeutics Inc.	1
Jerry Vockley, MD, PhD	1
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Sponsor Type

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Sponsor Type for OLPRUVA
Sponsor	Trials
Other	1
Industry	1
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