Azathioprine in MOGAD

• Status: Not yet recruiting
• Trial end date: 2028-10-01
• Target enrollment: 0
• Participant gender: All

Summary

MOG-IgG associated disease (MOGAD) is a rare inflammatory disease of the central nervous system recently described. Initially reported as monophasic, data from incident cohorts suggests that around 50% of adult patients with MOG-Ab may relapse within the first two years of the disease, with most of relapses occurring early after disease onset.

No randomized controlled trial has ever been performed and therapeutic guidelines for this disease remain unclear especially after a single event. In short-sized and mainly retrospective study, azathioprine, an immunosuppressant drug, have showed promising results on preventing the risk of relapse in MOGAD patients.

The hypothesis is that the initiation of a treatment after a first attack of MOGAD should prevent further relapse and disability accrual. The investigators propose herein the first randomized controlled trial in MOGAD, to evaluate the efficacy of azathioprine to prevent relapses, after a first attack, in a placebo double-blinded design.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hospices Civils de Lyon
• Treatments: Azathioprine

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

Exclusion Criteria:

• Hypersensitivity to azathioprine or steroids

• Active infections or cancer

• Seriously impaired hepatic or bone marrow functions:

Lymphocyte count < 1000/ml and or Polynuclear neutrophil count < 1500/ml ALT and/or AST > 3N

• Any live vaccine in the past 3 months

• 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 allopurinol and febuxostat

• Necessary use of any another immunosuppressive therapy different than azathioprine or steroids

• Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy is use. 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.

• Female subjects who have a positive blood pregnancy test result, are pregnant or are currently breast feeding. All female subjects of childbearing potential must practice effective contraception during the study.

• Inability to comply with study requirements

• Vulnerable patients (defined by articles L1121-5 to L1121-8 and L1122-1-2 from French Public Health Code)