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Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels

NCT05241262 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-01-23

No results posted yet for this study

Summary

N-Acetylcysteine (NAC), an anti-oxidant, will be studied to investigate the effects on brain glutathione levels, cognitive skills, motor skills, and quality of life.

A group of 18 participants will take either 1800, 3600 or 5400 mg per day of N-acetylcysteine (NAC) for 3 months in this dose escalation study. The investigators want to determine first if the 3600 mg dose per day is safe and might provide some efficacy. If the 3600 mg dose is safe, then additional participants will be treated with 5400 mg per day of NAC, for up to a total of 18 participants. If the 3600 mg per day dose is unsafe, then participants will be treated with the 1800 mg per day dose. Data from this pilot study will be used to determine the most safe and effective dose of NAC for a future clinical trial.

Conditions

Interventions

DRUG

N-Acetylcysteine

1800, 3600 or 5400 mg NAC per day (divided into 3 daily doses) depending on dose toxicity plan

Sponsors & Collaborators

  • United States Department of Defense

    collaborator FED

  • Michio Hirano, MD

    lead OTHER

Principal Investigators

  • Michio Hirano, MD · Columbia University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-07-06
Primary Completion
2026-03-31
Completion
2026-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05241262 on ClinicalTrials.gov