Can a common antioxidant help mitochondrial disease?
NCT ID NCT05241262
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.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Columbia University Irving Medical Center
RECRUITINGNew York, New York, 10032, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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