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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)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4 months

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4 months

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Maximum tolerated dose (MTD) of NAC

Secondary outcome measures

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

Headache

Nausea

Diarrhea

Abdominal discomfort

Dyspepsia

Pruritis

Arthropod bite

Road traffic accident

Alcohol abuse

Cellulitis

Gastrointestinal disorder

Abdominal pain

Vomiting

Reflux disease

Dizziness

Insomnia

Rash

Energy increased

Groin Abscess

Flushing

Fatigue

Panic attack

Depression

Blood pressure increased

100%

80%

60%

40%

20%

Study treatment Arm

NAC Plus CM

Placebo Plus CM

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

0 / 2Eligibility criteria met

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

865 Previous Clinical Trials

327,681 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the aggregate figure of participants enrolled in this trial?

"Affirmative. The clinicaltrials.gov website displays that this medical investigation is actively recruiting participants; the trial was first published on December 1st of 2022 and last updated on November 10th, 2022. A total of 18 study subjects are needed for enrollment at a single site."

Answered by AI

Is N-Acetylcysteine safe for consumption by individuals?

"Owing to limited data regarding its safety and efficacy, N-Acetylcysteine was assigned a score of 1 as per our team's assessment at Power."

Answered by AI

Is enrollment limited to those over 70 years of age for this clinical trial?

"This clinical trial is accepting volunteers who are of legal age and under 80 years old."

Answered by AI

What criteria must a person fulfill in order to qualify for this clinical trial?

"The scope of this particular study involves enrolling 18 persons with mitochondrial diseases aged between 18 and 80. The vital requirements for enrolment are as follows: Ages 18-80 years, Deficiency in brain glutathione (GSH) ascertained by magnetic resonance spectroscopic imaging (MRSI), Carrying the m.3243A>G mtDNA mutation or suspected to possess it (genetic confirmation is mandatory before NAC administration)."

Answered by AI

Are there opportunities for enrolment into this clinical experiment currently?

"Affirmative, information provided on clinicaltrials.gov confirms that this medical research is currently recruiting volunteers. This trial was initially posted on December 1st 2022 and the most recent update took place on November 10th 2022 - 18 participants are needed from a single study site."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

Michio Hirano, MD 2023. "Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05241262.

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