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

NCT ID: NCT04542161

Last Updated: 2025-03-20

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 95 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-09-01
• Study Completion Date: 2026-04-30

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.

Detailed Description

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.

Conditions

Chronic Fatigue Syndrome; Myalgic Encephalomyelitis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: OTHER
• Blinding Strategy: DOUBLE

Study Groups

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

Group Type: ACTIVE_COMPARATOR

NAC 900mg/day

Intervention Type: DRUG

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

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

Group Type: ACTIVE_COMPARATOR

NAC 3600mg/day

Intervention Type: DRUG

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

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

Group Type: PLACEBO_COMPARATOR

NAC 0mg/day (Placebo)

Intervention Type: DRUG

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

Interventions

NAC 900mg/day

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

Intervention Type: DRUG

NAC 3600mg/day

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

Intervention Type: DRUG

NAC 0mg/day (Placebo)

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

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 21 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

Weill Medical College of Cornell University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Dikoma C. Shungu, Ph.D.

Role: PRINCIPAL_INVESTIGATOR

Weill Medical College of Cornell University

Locations

Weill Cornell Medicine

New York, New York, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Xiangling Mao, MS

Role: CONTACT

xim2004@med.cornell.edu

2127462632

Other Identifiers

R01NS116887

Identifier Type: NIH

Identifier Source: secondary_id

View Link

20-01021280

Identifier Type: -

Identifier Source: org_study_id