Mitochondrial Disease study: N-Acetylcysteine
Official title Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
ClinicalTrials.gov ID: NCT05241262
What this study is testing
What is N-Acetylcysteine?
An investigational oral medication being studied for mitochondrial disease.
Also referred to as NAC.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study wants to find out if taking NAC can safely raise brain glutathione levels and improve thinking, movement, and quality of life in people with a specific mitochondrial mutation.
- Who it's for
- People who have the m.3243A>G mitochondrial mutation and low brain glutathione levels.
- What's involved
- Participants will take a daily dose of NAC (1800, 3600, or 5400 mg) for 3 months, with doses adjusted based on safety results. They will have regular visits and tests to monitor safety and changes in brain chemistry and function.
- Time commitment
- The study lasts 3 months, with several clinic visits for dosing, safety checks, and assessments of brain glutathione, cognition, motor skills, and quality of life.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 80
You may be able to join if
- 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...
You likely can't join if
- 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
The study team makes the final eligibility decision.
Where it is enrolling
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at New York, New York, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.