Assessment of N-Acetylcysteine as Therapy for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome

Study Purpose

Chronic fatigue syndrome/myalgic encephalomyelitis (ME/CFS) is an unexplained multisymptom/multisystem disorder for which there are currently no validated treatments. The present exploratory clinical trial aims to advance our understand of the mechanisms of in situ GSH synthesis control through assessment of the response of brain GSH and plasma markers of oxidative stress to different doses of NAC in comparison to placebo, as a potential treatment for ME/CFS that would provide neuroprotection against oxidative stress by restoring cortical GSH reserves. If successful, this exploratory clinical trial would address a significant public health concern by shedding new light onto the mechanisms of action of NAC in brain GSH restoration, which could open a new avenue for the development of potentially effective treatments for a disorder, ME/CFS, that currently has none.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 21 Years - 60 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - Males or females, ages 21 to 60 years (inclusive).
  • - Baseline GSH levels at or less than a predefined cutoff value.
  • - Primary diagnosis of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS).
  • - Willing and capable of providing informed consent.

Exclusion Criteria:

  • - Significant and/or comorbid axis I (especially mood and anxiety) and axis II disorders.
  • - Any significant neurological illness or impairment.
  • - Other unstable medical conditions (asthma, hypertension, endocrine or metabolic disease, etc).
  • - History alcohol abuse.
  • - Positive urine toxicology at screening and on days of assessments.
  • - Positive pregnancy test at screening or on days of assessments.
  • - Contra-indication for clinical MRI scan (e.g., pacemaker, metallic prosthesis).
  • - Baseline GSH levels higher than a predefined cutoff value.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT04542161
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Weill Medical College of Cornell University
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Dikoma Shungu, Ph.D.
Principal Investigator Affiliation Weill Cornell Medicine
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

OtherNIH
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Chronic Fatigue Syndrome, Myalgic Encephalomyelitis
Additional Details

This phase two, single-site study will utilize a double-blind, placebo-controlled, randomized, pre-/post-treatment design to investigate the effect of NAC dosing on brain GSH levels and measure temporally concordant plasma levels of several established circulating markers of oxidative stress. Three study groups, of 20 subjects each (for a total of 60 who completed all components of the study), will each be administered a different dose (0 mg/day, 900mg/day, 3600mg/day) of the study intervention over a four week period; N-acetylcysteine (NAC) treatment. Subjects receiving 0 mg/day dose will be administered a placebo. Baseline visit assessments will include blood collection, survey questionnaires, MRI and MRS imaging. Subjects whose initial screening confirms low GSH level at baseline will be provided with a 4-week supplement of anonymized NAC or placebo caplets. After 4 weeks, subjects will then undergo a follow-up visit to repeat the baseline assessments.

Arms & Interventions

Arms

Active Comparator: NAC 900mg/day

Subjects who pass screening may be randomly assigned to this arm where they will self administer NAC 900mg/day caplets for a four week period

Active Comparator: NAC 3600mg/day

Subjects who pass screening may be randomly assigned to this arm where they will self administer NAC 3600mg/day caplets for a four week period

Placebo Comparator: NAC 0mg/day (Placebo)

Subjects who pass screening may be randomly assigned to this arm where they will self administer NAC 0mg/day (placebo) caplets for a four week period

Interventions

Drug: - NAC 900mg/day

self administer NAC 900mg/day caplets for a four week period

Drug: - NAC 3600mg/day

self administer NAC 3600mg/day caplets for a four week period

Drug: - NAC 0mg/day (Placebo)

self administer NAC 0mg/day (placebo) caplets for a four week period

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Weill Cornell Medicine, New York, New York

Status

Recruiting

Address

Weill Cornell Medicine

New York, New York, 10021