Comparing immediate and delayed treatments for children with anti-MOG antibody-related acute demyelinating syndromes
Immediate Versus Delayed Treatment With Azathioprine or Rituximab in Anti-myelin Oligodendrocytes Glycoprotein (Anti-MOG) Antibodies Associated Acute Demyelinating Syndromes in Children: a Randomized Controlled Clinical Trial
This study is testing whether starting treatment right away with Azathioprine or Rituximab can help children with anti-MOG antibody-related acute demyelinating syndromes do better in the long run compared to waiting for treatment.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 86 (estimated) |
| Ages | 6 Years to 17 Years |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Drugs / interventions | rituximab, natalizumab, daclizumab, methotrexate |
| Locations | 9 sites (Besançon and 8 other locations) |
| Trial ID | NCT05545384 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effects of immediate versus delayed treatment with Azathioprine or Rituximab in children diagnosed with acute demyelinating syndromes associated with anti-MOG antibodies. The study aims to determine whether early intervention can reduce long-term neurological sequelae and the frequency of relapses compared to standard care. Children aged 6 to 18 years with confirmed anti-MOG antibodies and specific acute neurological symptoms will be enrolled. The trial will assess the efficacy of these treatments in improving patient outcomes over time.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 6 to 18 years with confirmed anti-MOG antibodies and acute neurological symptoms lasting more than 24 hours.
Not a fit: Patients with uncontrolled infections, prior allergies to the study medications, or other specific exclusion criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved long-term neurological outcomes and reduced disability for children affected by acute demyelinating syndromes.
How similar studies have performed: While the approach of using Azathioprine and Rituximab in this context is novel, previous studies have shown promising results in treating similar conditions with these medications.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Children \< 18 years old and ≥ 6 years old at baseline * Children weight ≥ 20 kg * All ADS with confirmed anti-MOG-Abs at onset including any acute neurologic symptom with a duration of more than 24H of inflammatory causes (including optic neuritis, transverse myelitis, rhombencephalitis, ADEM, NMOSD) Without any previous treatment other than steroids * Informed consent signed by both parents and the child * Expanded Disability Status Scale (EDSS) \< 5.5 * Affiliated to French social security regime Exclusion Criteria: * Current infection with SARS-COV2 (positive PCR) * Any prior allergy to azathioprine or rituximab with hypersensitivity to active substances, murine proteins or to any of the excipients. * Any prior history of uncontrolled cancer during the last 2 years * Uncontrolled infections (Hepatitis B, C and HIV) * Any prior history of cardiac dysfunction and/or hypertension * Any progressive or non-relapsing form demyelinating diseases * Any previous treatment with natalizumab, daclizumab, fingolimod, methotrexate, cyclosporine, mycophenolate mofetil, rituximab in the last 6 months, or determined by the treating physician to have residual immune suppression from these or other immunosuppressive treatments * CD4+, CD8+, or CD19+ absolute cell count, wbc, neutrophiles in blood at screening below lower limits of normal (LLN) * Creatinine\>30µmol/L * Platelets \<70 000mm3 * Haemoglobin \< 8g/dL * Acute renal insufficiency (clearance \< 30 ml/min) * Prior documented history of hemostase perturbation (TP and/or TCA more than twice of the witnesse's TP and/or TCA) * Prior documented history of increased liver enzyme level (ASAT and/or ALAT) \> 2N. * TP \<70% * Total bilirubin \> 2N * Any patient with allopurinol treatment and immunosupressive treatment with concomitant use of xanthine oxidase inhibitors (e.g. allopurinol, oxipurinol/thiopurinol, febuxostat) * Patients with two inactive TPMT or NUDT15 alleles (homozygous deficient or double heterozygote) * Pregnancy or lactating woman or wish for future pregnancy * Refusal to have a highly effective contraception during traitment and for one year (12 months) after the end of the experimental treatment * participation to another interventional study within 5 half-lives prior to baseline. * Active, severe infections (including tuberculosis, HBV and HCV, HIV, herpes, VZV, EBV and CMV) * Psychosis not controlled by treatment * Patients with Lesch Nyhan syndrome * Pheochromocytoma * Scleroderma * Untreated peptic ulcer * Myasthenia gravis * Any other medical illness or disability that, in the opinion of the investigator, would compromise effective trial participation
Where this trial is running
Besançon and 8 other locations
- CHU Besançon — Besançon, France (Not_yet_recruiting)
- CHU Bordeaux — Bordeaux, France (Not_yet_recruiting)
- CHU Brest — Brest, France (Not_yet_recruiting)
- HCL de Bron — Bron, France (Not_yet_recruiting)
- Hôpital Bicêtre — Le Kremlin-Bicêtre, France (Recruiting)
- CHRU Lille — Lille, France (Not_yet_recruiting)
- CHU Monptellier — Montpellier, France (Not_yet_recruiting)
- CHU Strasbourg — Strasbourg, France (Not_yet_recruiting)
- CHU Toulouse Purpan — Toulouse, France (Not_yet_recruiting)
Study contacts
- Study coordinator: Kumaran DEIVA, phD
- Email: kumaran.deiva@aphp.fr
- Phone: 0145213112
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.