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Can a common antioxidant help mitochondrial disease?

NCT ID NCT05241262

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 18 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2023

Expected to finish

Mar 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Ages 18-80 years * Low brain glutathione (GSH) levels as determined by magnetic resonance spectroscopic imaging (MRSI) * Individuals who carry, or are suspected of carrying the m.3243A\>G mitochondrial mutation (genetic confirmation of mutation required prior to initiation of NAC) Exclusion Criteria: * Individuals with normal brain glutathione levels * Pregnant or lactating individuals * Medically unstable as determined by the Principal Investigator * Allergy to NAC or other sulfur-containing drug * Inability to adhere to study protocol

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Columbia University Irving Medical Center

    RECRUITING

    New York, New York, 10032, United States

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