← Back to Search

Antioxidant

N-Acetylcysteine for Mitochondrial Disease

Phase 1
Recruiting
Led By Darryl DeVivo, MD
Research Sponsored by Michio Hirano, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ages 18-80 years
Individuals who carry, or are suspected of carrying the m.3243A>G mitochondrial mutation (genetic confirmation of mutation required prior to initiation of NAC)
Must not have
Allergy to NAC or other sulfur-containing drug
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 4 months
Awards & highlights
No Placebo-Only Group

Summary

This trial will study the effects of N-acetylcysteine (NAC), an anti-oxidant, on brain glutathione levels, cognitive skills, motor skills, and quality of life. 18 participants will take either 1800, 3600 or 5400 mg per day of NAC for 3 months. The goal is to determine a safe and effective dose of NAC for a future clinical trial.

Who is the study for?
This trial is for adults aged 18-80 with low brain glutathione levels and the m.3243A>G mitochondrial mutation. Participants must be able to follow the study protocol and not be pregnant, lactating, allergic to NAC or sulfur drugs, or medically unstable.
What is being tested?
The study tests different doses of N-Acetylcysteine (NAC) on brain health and quality of life in people with a specific mitochondrial disease. It starts at 3600 mg/day to check safety and effectiveness before possibly increasing to 5400 mg/day or decreasing to 1800 mg/day based on results.
What are the potential side effects?
Potential side effects of N-Acetylcysteine may include gastrointestinal discomfort, rash, headache, dry mouth, dizziness, or an allergic reaction for those sensitive to sulfur-containing drugs.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am between 18 and 80 years old.
Select...
I carry or might carry the m.3243A>G mitochondrial mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I am allergic to NAC or sulfur drugs.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~4 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Maximum tolerated dose (MTD) of NAC
Secondary study objectives
Change in Brain glutathione (GSH) level
Change in Functional Exercise Capacity (6 minute walk test (6MWT))
Change in Global Neuropsychological Score
+2 more

Side effects data

From 2015 Phase 3 trial • 302 Patients • NCT01675661
7%
Headache
5%
Nausea
5%
Diarrhea
2%
Abdominal discomfort
2%
Dyspepsia
2%
Pruritis
1%
Cellulitis
1%
Alcohol abuse
1%
Road traffic accident
1%
Arthropod bite
1%
Gastrointestinal disorder
1%
Abdominal pain
1%
Vomiting
1%
Reflux disease
1%
Dizziness
1%
Insomnia
1%
Rash
1%
Energy increased
1%
Groin Abscess
1%
Flushing
1%
Fatigue
1%
Panic attack
1%
Depression
1%
Blood pressure increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
NAC Plus CM
Placebo Plus CM

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Active drug (NAC)Experimental Treatment1 Intervention
Participants will receive NAC for 3 months.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
N-Acetylcysteine
2013
Completed Phase 3
~1470

Find a Location

Who is running the clinical trial?

Michio Hirano, MDLead Sponsor
1 Previous Clinical Trials
Darryl C. De Vivo, MDLead Sponsor
United States Department of DefenseFED
910 Previous Clinical Trials
333,713 Total Patients Enrolled

Media Library

N-Acetylcysteine (Antioxidant) Clinical Trial Eligibility Overview. Trial Name: NCT05241262 — Phase 1
Mitochondrial Disease Research Study Groups: Active drug (NAC)
Mitochondrial Disease Clinical Trial 2023: N-Acetylcysteine Highlights & Side Effects. Trial Name: NCT05241262 — Phase 1
N-Acetylcysteine (Antioxidant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05241262 — Phase 1
~4 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top