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

Part of paid clinical trials in New York, New York.

Sponsor
Michio Hirano, MD
Study ID
NCT05241262
Phase
PHASE1
Status
Recruiting
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Conditions

  • Mitochondrial Disease

Eligibility Criteria

Sex
ALL
Age
18 Years - 80 Years
Healthy Volunteers
Not accepted

Interventions

  • N-Acetylcysteine — DRUG
    1800, 3600 or 5400 mg NAC per day (divided into 3 daily doses) depending on dose toxicity plan

Study Details

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.

Key Dates

Start date
Jul 6, 2023
Status verified
Jan 2026
Primary completion
Mar 31, 2026
Completion
Mar 31, 2026

Study Design

Enrollment
18 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Active drug (NAC)
    Participants will receive NAC for 3 months.

Primary Outcome Measure

Maximum tolerated dose (MTD) of NAC [ Time Frame: 4 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Columbia University Irving Medical CenterNew YorkNew York10032
Kris Engelstad, MS, CGC
[email protected]
212-342-5767
Michio Hirano, MD (PRINCIPAL_INVESTIGATOR)

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By research site
Columbia University Irving Medical Center· New York, NY

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