NCT04542161

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

Mechanistic Assessment of N-Acetylcysteine as an Antioxidant Therapy for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) Through Dose Response and Treatment Target Engagement

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 95 (estimated)
• Sponsor: Weill Medical College of Cornell University · Academic / Other
• Sex: All
• Age: 21 Years – 60 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
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

Timeline

• Start date: 2020-09-01
• Primary completion: 2026-04-30
• Completion: 2026-04-30
• First posted: 2020-09-09
• Last updated: 2026-02-25

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study