Safety and Efficacy of Tocilizumab in Patients With Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease

NCT ID: NCT06452537

Last Updated: 2026-01-08

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 102 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-07-09
• Study Completion Date: 2026-07-01

Brief Summary

The purpose of the study is to evaluate the safety and efficacy of Tocilizumab in MOGAD.

Detailed Description

Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is a rare autoimmune disease of the central nervous system, which can cause optic neuritis, myelitis, brainstem encephalitis, or encephalitis. The specific autoantibody against myelin oligodendrocyte glycoprotein antibody (MOG-IgG) has been indicated to contribute to the pathogenesis of the disease. Data from several cohorts suggests that around 50% of adult patients with MOG-IgG may relapse within the first two years of the disease, with most of relapses occurring early after disease onset. Few randomized controlled trials have ever been performed and therapeutic guidelines for this disease remain unclear especially after a single event. There is no drug approved for MOGAD by FDA. IL-6 is a pro-inflammatory cytokine which can promotes B cell activation, blood-brain barrier dysfunction, leukocyte migration, and the production of autoantibodies. Tocilizumab (ACTEMRA®), a humanized monoclonal antibody against the IL-6 receptor, has shown beneficial clinical effects and reduction of the risk of relapses in some patients with MOGAD. However, the efficacy of tocilizumab in MOGAD warrants further clinical trials.

Conditions

Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Tocilizumab with oral prednisone

Tocilizumab will be intravenously administered as the dosage of 8 mg/kg every 4 weeks, with oral prednisone

Group Type: EXPERIMENTAL

Tocilizumab

Intervention Type: DRUG

Tocilizumab will be intravenously administered as the dosage of 8 mg/kg every 4 weeks, with oral prednisone.

Prednisone

Intervention Type: DRUG

Prednisone tapering protocol : If the starting dose is over 20mg/day, then reduce by one tablet weekly. Until the dose is reduced to 20mg/day then 20mg/day for two weeks→17.5mg/day for two weeks→12.5mg for four weeks→10mg for four weeks→7.5mg as a maintain dosage

Prednisone

Group Type: EXPERIMENTAL

Prednisone

Intervention Type: DRUG

Prednisone tapering protocol : If the starting dose is over 20mg/day, then reduce by one tablet weekly. Until the dose is reduced to 20mg/day then 20mg/day for two weeks→17.5mg/day for two weeks→12.5mg for four weeks→10mg for four weeks→7.5mg as a maintain dosage

Interventions

Tocilizumab

Tocilizumab will be intravenously administered as the dosage of 8 mg/kg every 4 weeks, with oral prednisone.

Intervention Type: DRUG

Prednisone

Prednisone tapering protocol : If the starting dose is over 20mg/day, then reduce by one tablet weekly. Until the dose is reduced to 20mg/day then 20mg/day for two weeks→17.5mg/day for two weeks→12.5mg for four weeks→10mg for four weeks→7.5mg as a maintain dosage

Intervention Type: DRUG

Other Intervention Names

ACTEMRA®

Eligibility Criteria

Inclusion Criteria

1. Participants who are aged ≥12 years at the time of signing Informed Consent Form

2. Confirmed diagnosis of MOGAD with a history of ≥1 MOGAD relapse in the 12 months prior to screening or ≥2 attacks in the 24 months prior to screening

3. Anti-MOG antibody seropositive

4. For women of childbearing potential: participants who agree to remain abstinent or use adequate contraception during the treatment period and for at least 3 months after the final dose of tocilizumab

5. Patients must give written informed consent

Exclusion Criteria

1. Any concomitant disease other than MOGAD that may require treatment with oral immunosuppressants or prednisone at doses >20 mg/day (or equivalent)

2. Receipt of the following at any time prior to randomization Alemtuzumab Total lymphoid irradiation Bone marrow transplant T-cell vaccination therapy Receipt of rituximab or any experimental B-cell depleting agent within 6 months prior screening and B-cells below the lower limit of normal.

Receipt of intravenous immunoglobulin (IVIG) or plasma exchange (PE) within 1 month prior to randomization.

Receipt of any of the following within 3 months prior to randomization:

Natalizumab (Tysabri®). Methotrexate Mitoxantrone Cyclophosphamide Eculizumab

Receipt of any of the following within 6 weeks prior to randomization:

Tacrolimus Cyclosporin Mycophenolate mofetil

3. Participants who are pregnant or breastfeeding, or intending to become pregnant during the study or within 3 months after the final dose of tocilizumab

4. Participants with active or presence of recurrent bacterial, viral, fungal, mycobacterial infection, or other infection at baseline

5. Participants with evidence of latent or active tuberculosis (excluding patients receiving chemoprophylaxis for latent tuberculosis infection)

6. Participants with positive screening tests for hepatitis B and C

7. Receipt of live or live attenuated vaccine within 6 weeks prior to baseline

8. Known history of a severe allergy or reaction to any biologic therapy.

9. History of alcohol, drug, or chemical abuse, or a recent history of such abuse < 1 year prior to randomization

10. WBC < 3.0 × 10^3/mL, ANC < 2.0 × 10^3/mL, PLT < 10 × 10^4/mL, AST or ALT>1.5 ×ULN, Lymphocyte count < 0.5 × 10^3/mL

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tianjin Medical University General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Chao Zhang

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Chao Zhang, M.D., Ph.D

Role: PRINCIPAL_INVESTIGATOR

Tianjin Medical University General Hospital

Locations

Anhui Provincial Hospital

Hefei, Anhui, China

The Second Hospital of Lanzhou University

Lanzhou, Gansu, China

Hebei Children's Hospital

Shijiazhuang, Hebei, China

The Second Hospital of Hebei Medical University

Shijiazhuang, Hebei, China

The Second Affiliated Hospital of Harbin Medical University

Harbin, Heilongjiang, China

The First Affiliated Hospital of Zhengzhou University

Zhenzhou, Henan, China

Ordos Central Hospital

Ordos, Inner Mongolia, China

The Third Affiliated Hospital of Soochow University

Changzhou, Jiangsu, China

Children's Hospital of Soochow University

Suzhou, Jiangsu, China

Affiliated Hospital of Xuzhou Medical University

Xuzhou, Jiangsu, China

General Hospital of Ningxia Medical University

Yinchuan, Ningxia, China

First Hospital of Shanxi Medical University

Taiyuan, Shanxi, China

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, China

People's Hospital of Xinjiang Uygur Autonomous Region

Ürümqi, Xinjiang Uygur Autonomous Region, China

Beijing Tiantan Hospital, Capital Medical University

Beijing, , China

China-Japan Friendship Hospital

Beijing, , China

Chinese PLA General Hospital

Beijing, , China

The First Affiliated Hospital of Tsinghua University

Beijing, , China

XuanWu Hospital Capital Medical University

Beijing, , China

Huashan Hospital, Fudan University

Shanghai, , China

Tianjin Huanhu Hospital

Tianjin, , China

Tianjin Children's Hospital

Tianjin, , China

Countries

China

Other Identifiers

2024035

Identifier Type: -

Identifier Source: org_study_id