Can a common antioxidant help mitochondrial disease?

NCT ID NCT05241262

Recruiting now Disease control Sponsor: Michio Hirano, MD Source: ClinicalTrials.gov ↗

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

Summary

This phase 1 trial tests N-acetylcysteine (NAC), an antioxidant, in 18 adults with a specific mitochondrial disease caused by the m.3243A>G mutation and low brain glutathione levels. Participants take one of three daily doses (1800, 3600, or 5400 mg) for 3 months to find the safest dose and see if it improves brain health, thinking, movement, and quality of life.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: N-acetylcysteine (NAC)
What this could lead to: If successful, this could point toward a treatment to improve brain function and quality of life for people with this mitochondrial disease.
What could go wrong: This is a very early, small phase 1 trial with only 18 participants. It primarily tests safety and dosing, not effectiveness. The benefits are uncertain.

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Conditions

The condition(s) this trial relates to.

Deafness diabetes mellitus inborn mitochondrial metabolism disorder MELAS syndrome mitochondrial disease

As listed by the trial registrant

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

MITOCHONDRIAL DISEASE

Contacts and locations

Locations

Columbia University Irving Medical Center
RECRUITING

New York, New York, 10032, United States

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