Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
Status:
Recruiting
Trial end date:
2024-06-01
Target enrollment:
Participant gender:
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 patients will take either 1800, 3600 or 5400 mg per day of NAC (for 3 months)
for a dose escalation study; this means that we 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 we will
treat additional patients with 5400 mg per day of NAC up to a total of 18 patients. 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.