Neuroprotection With N-acetyl Cysteine for Patients With Progressive Multiple Sclerosis

NCT05122559 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: P0549747
• Study Type: interventional
• Target: 98
• Locations: 1 country
• Sites: 1

Brief Summary

This study evaluates the effectiveness of N-acetyl cysteine (NAC) in the treatment of progressive multiple sclerosis. Half of the patients will receive NAC, while the other half will receive a placebo.

Conditions

Multiple Sclerosis; Multiple Sclerosis, Primary Progressive; Multiple Sclerosis, Secondary Progressive

Outcome Measures

Primary Outcomes (2)

• Safety and tolerability

  Number of adverse events recorded by system, severity, and by relationship to treatment arm.

  15 months

• Effect of NAC on on progression of brain, thalamic and cervical cord atrophy

  The primary endpoint is brain, thalamic and cord atrophy measured by brain and cervical spine MRI at month 3 and month 15.

  15 months

Secondary Outcomes (1)

• Clinical effects of NAC

  15 months

Other Outcomes (1)

• Effect of NAC on progression of MS

  15 months

Study Arms (2)

N-acetyl cysteine

ACTIVE COMPARATOR

N-acetyl cysteine (NAC) 1200mg t.i.d.

Drug: N-acetyl cysteine

Placebo

PLACEBO COMPARATOR

Placebo 1200mg t.i.d.

Drug: Placebo

Interventions

Placebo DRUG

Lactose Monohydrate, USP (100%), magnesium stearate, silicon dioxide NF

Placebo

N-acetyl cysteine DRUG

N-acetyl cysteine (NAC) is a Glutathione (GSH) precursor with antioxidant properties which make it relevant for neuroprotection.

Also known as: NAC

N-acetyl cysteine

Eligibility Criteria

• Age: 40 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \- 40-70 (inclusive) years in age,
• meet 2017 McDonald criteria (Thompson 2018),
• patients with primary or secondary progressive MS (Thompson 2018),
• at least 2 years since progressive symptom onset,
• evidence of clinical changes over the previous 2 years unrelated to relapses: increased EDSS or 20% slowing on 25-foot walk, change of ambulatory support, cognitive change documented on cognitive testing. Progression defined by patients in terms of ambulation perimeter or type of support to ambulate are acceptable if aforementioned physician-based measure changes are not available.
• EDSS score 3.0 to 7.0 (inclusive),
• can be on a stable disease-modifying treatment initiated \> 3 months prior to screening,
• can be on stable doses of dalfampridine initiated at least one month before screening.

You may not qualify if:

• \- MS relapses in the previous 6 months
• oral glucocorticosteroid treatment within the prior 3 months
• patient with issues undergoing MRI scans
• pregnancy or breastfeeding
• women of child-bearing potential not able to utilize an effective form of contraception for the duration of the study
• history of bleeding disorders
• active gastrointestinal ulcers
• abnormal liver function testing (aminotransferase (AST) or alanine aminotransferase (ALT) \>2 times upper limit of normal)
• current treatment for active malignancy or metastatic malignancy treated in the past year
• alcohol or substance use disorder
• allergy to NAC
• planned surgery or move within 15 months
• use of medications/supplements with antioxidant properties (including over-the-counter NAC)

Sponsors & Collaborators

• Emmanuelle Waubant, MD PhD: lead
• United States Department of Defense: collaborator

Study Sites (1)

University of California, San Francisco

San Francisco, California, 94158, United States

Location

Related Publications (1)

• Schoeps VA, Graves JS, Stern WA, Zhang L, Nourbakhsh B, Mowry EM, Henry RG, Waubant E. N-Acetyl Cysteine as a Neuroprotective Agent in Progressive Multiple Sclerosis (NACPMS) trial: Study protocol for a randomized, double-blind, placebo-controlled add-on phase 2 trial. Contemp Clin Trials. 2022 Nov;122:106941. doi: 10.1016/j.cct.2022.106941. Epub 2022 Sep 28.

  PMID: 36182028 DERIVED

MeSH Terms

Conditions

Multiple Sclerosis; Multiple Sclerosis, Chronic Progressive

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor, Neurology UCSF Weill Institute for Neurosciences

Study Record Dates

• First Submitted: November 8, 2021
• First Posted: November 16, 2021
• Study Start: February 16, 2022
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027
• Last Updated: March 11, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)