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

NCT04542161 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 20-01021280R01NS116887
• Study Type: interventional
• Target: 95
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Myalgic Encephalomyelitis; Chronic Fatigue Syndrome

Outcome Measures

Primary Outcomes (2)

• Change in GSH levels of treatment response: measure 1

  Levels of occipital cortex GSH, as measured in vivo with 1H MRS

  pre/post 4 weeks of NAC supplementation

• Change in GSH levels of treatment response: measure 2

  Levels of striatal GSH, as measured in vivo with 1H MRS

  pre/post 4 weeks of NAC supplementation

Secondary Outcomes (8)

• Change of levels of ventricular CSF lactate of treatment response

  pre/post 4 weeks of NAC supplementation

• Change of regional cerebral blood flow (rCBF) of treatment response

  pre/post 4 weeks of NAC supplementation

• Change in Oxidative stress levels of treatment response: measure 1

  pre/post 4 weeks of NAC supplementation

• Change in Oxidative stress levels of treatment response: measure 2

  pre/post 4 weeks of NAC supplementation

• Change in Oxidative stress levels of treatment response: measure 3

  pre/post 4 weeks of NAC supplementation

Study Arms (3)

NAC 900mg/day

ACTIVE COMPARATOR

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

Drug: NAC 900mg/day

NAC 3600mg/day

ACTIVE COMPARATOR

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

Drug: NAC 3600mg/day

NAC 0mg/day (Placebo)

PLACEBO COMPARATOR

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

Drug: NAC 0mg/day (Placebo)

Interventions

NAC 900mg/day DRUG

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

NAC 900mg/day

NAC 3600mg/day DRUG

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

NAC 3600mg/day

NAC 0mg/day (Placebo) DRUG

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

NAC 0mg/day (Placebo)

Eligibility Criteria

• Age: 21 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Weill Medical College of Cornell University: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (1)

Weill Cornell Medicine

New York, New York, 10021, United States

RECRUITING

MeSH Terms

Conditions

Fatigue Syndrome, Chronic

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Encephalomyelitis; Neuroinflammatory Diseases; Nervous System Diseases; Neuromuscular Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Dikoma C. Shungu, Ph.D.

  Weill Medical College of Cornell University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiangling Mao, MS

CONTACT

2127462632; xim2004@med.cornell.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 28, 2020
• First Posted: September 9, 2020
• Study Start: September 1, 2020
• Primary Completion: April 30, 2026
• Study Completion: April 30, 2026
• Last Updated: February 25, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)