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Using azathioprine to prevent relapses in MOGAD

A Randomized, Placebo-controlled Phase 3 Trial of Azathioprine for the Prevention of Relapse in Myelin-oligodendrocyte-glycoprotein (MOG)-Antibody Associated Disease

Phase 3 Interventional Hospices Civils de Lyon · NCT05349006

This study is testing if the medication azathioprine can help prevent relapses in people with MOG-IgG associated disease after their first attack.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment126 (estimated)
Ages18 Years and up
SexAll
SponsorHospices Civils de Lyon Academic / other
Locations17 sites (Bordeaux and 16 other locations)
Trial IDNCT05349006 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the efficacy of azathioprine, an immunosuppressant, in preventing relapses of MOG-IgG associated disease (MOGAD) after a patient's first attack. It is the first randomized controlled trial for this rare inflammatory condition of the central nervous system, which has shown promising results in smaller studies. Participants will be randomly assigned to receive either azathioprine or a placebo in a double-blind manner. The study aims to clarify treatment guidelines for MOGAD, which currently remain uncertain.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older who have experienced their first documented acute demyelinating syndrome attack within the past three months and tested positive for MOG antibodies.

Not a fit: Patients with hypersensitivity to azathioprine, active infections, or serious bone marrow dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the risk of relapse and disability in patients with MOGAD.

How similar studies have performed: While this is the first randomized controlled trial for MOGAD, previous smaller studies have indicated potential benefits of azathioprine in preventing relapses.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Age ≥ 18 years
* First attack of documented acute demyelinating syndrome of the central nervous system, within the past 3 months, whatever the severity or the clinical phenotype
* Tested positive for MOG-Ab, confirmed in a centralized lab (Lyon referral centre)
* Ability of the subject to understand the purpose and risks of the study and provide signed and dated written informed consent.
* Patients should be beneficiary of health care coverage under the social security system
* Female patients of childbearing potential should have effective contraception throughout the course of treatment and for at least three months after stopping treatment.

Exclusion Criteria:

* Hypersensitivity to azathioprine or steroids
* Active infections or cancer (including tuberculosis, hepatitis, herpes and VZV)
* Psychosis not controlled by treatment
* Seriously impaired bone marrow functions: Lymphocyte count \< 1000/ml and or Polynuclear neutrophil count \< 1500/ml
* Seriously impaired hepatic functions: ALT and/or AST \> 3N
* Seriously impaired renal functions: GFR \< 29 ml/min/1.73m²
* Any live vaccine in the past 3 months or planned during the RCT and RCT+6months
* Thiopurine methyltransferase (TPMT) phenotype deficient or intermediate, with enzymatic activity \< 16 nmol/h/ml
* Unable to complete an MRI (e.g. due to pacemaker, severe claustrophobia, hypersensitivity to contrast media, or who lack adequate peripheral venous access)
* Necessary use of a xanthine oxidase inhibitor (Allopurinol, Oxipurinol, Thiopurinol, Febuxotat,…)
* Necessary use of angiotensin-converting-enzyme inhibitor, cotrimoxazole, cimetidine and indometacine
* Necessary use of an aminosalicylate derivates
* Necessary use of any another immunosuppressive therapy, different than azathioprine, or steroids
* Necessary use of cytotoxic therapy
* Necessary use of any other medical illness or disability that, in the opinion of the investigator, would compromise effective trial participation
* Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy is use within 5 half-lives prior to baseline. Participation in a non- interventional study can be allowed as long as this participation does not interfere with this protocol or is not likely to affect the subject's ability to comply with the protocol.
* For subjects coming back from strongyloidiasis endemic regions, a parasitology screening examination will be performed on faeces, and that appropriate treatment will be performed prior to administration of corticosteroids
* Patients with Lesch Nyhan syndrome
* Asian patients (probable mutation of the gene NUDT1)
* Female subjects who have a positive a positive urinary or blood pregnancy test result, are pregnant or are currently breast feeding
* Inability to comply with study requirements
* Person under legal protection or deprived of liberty

Where this trial is running

Bordeaux and 16 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Central Nervous System InflammationMOG-IgG Associated DiseaseMOGADazathioprineoptic neuritismyelitisneuromyelitis opticaMOG-IgG associated disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.