Actively Recruiting
Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
Led by Michio Hirano, MD · Updated on 2026-01-23
18
Participants Needed
1
Research Sites
138 weeks
Total Duration
On this page
Sponsors
M
Michio Hirano, MD
Lead Sponsor
U
United States Department of Defense
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ages 18-80 years
- Low brain glutathione levels confirmed by magnetic resonance spectroscopic imaging (MRSI)
- Individuals who carry or are suspected to carry the m.3243A>G mitochondrial mutation (genetic confirmation required before starting NAC)
You will not qualify if you...
- 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 drugs
- Inability to adhere to study protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
Research Team
K
Kris Engelstad, MS, CGC
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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