Using Tocilizumab to treat Generalized Myasthenia Gravis

A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of the Efficacy and Safety of Tocilizumab in the Treatment of Generalized Myasthenia Gravis

This study is testing if Tocilizumab can help people with Generalized Myasthenia Gravis who have anti-AChR antibodies feel better and improve their symptoms.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 64 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Tang-Du Hospital (Academic / other)
• Drugs / interventions: rituximab, tocilizumab, eculizumab, chemotherapy
• Locations: 6 sites (Beijing, Beijing Municipality and 5 other locations)
• Trial ID: NCT05067348 on ClinicalTrials.gov

What this trial studies

This is a randomized, double-blind, placebo-controlled study aimed at evaluating the efficacy and safety of Tocilizumab in patients diagnosed with Generalized Myasthenia Gravis who are positive for anti-AChR antibodies. Approximately 64 subjects will be enrolled across six study sites, with eligibility based on specific clinical classifications and scores. The study includes a randomized controlled period followed by an optional one-year open-label extension for participants who complete the initial phase.

Who should consider this trial

Good fit: Ideal candidates are patients diagnosed with Generalized Myasthenia Gravis who test positive for anti-AChR antibodies and meet specific clinical criteria.

Not a fit: Patients without anti-AChR or anti-MuSK antibodies, or those with clinically relevant active infections, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of Generalized Myasthenia Gravis, enhancing patients' quality of life.

How similar studies have performed: While this approach is being tested in this specific context, similar studies using Tocilizumab for autoimmune conditions have shown promising results.

Eligibility criteria

Inclusion Criteria:

1. Diagnosis of MG with anti-AChRantibody.
2. MGFA Clinical Classification Class II, III, or IV.
3. MG-ADL score of 5 or greater at screening and at randomization with \> 50% of this score attributed to non-ocular items.
4. QMG score of 11 or greater.
5. Subjects must be on:

   1. Cholinesterase inhibitor, with no dose increase within 4 weeks prior to randomization;
   2. Corticosteroids, with no dose increase within 4 weeks prior to randomization; or/and c. non-steroidal IST (including azathioprine, mycophenolate mofetil, tacrolimus, cyclosporine A), with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization.

Exclusion Criteria:

1. Participants had clinically relevant active infections (such as sepsis, pneumonia, or abscess) or severe infections (resulting in hospitalization or requiring antibiotic treatment) in the 4 weeks before randomization;
2. Those with a history of high-risk tuberculosis infection, acquired tuberculosis infection, and chronic hepatitis;
3. Human immunodeficiency virus (HIV) infection;
4. Thymomas that have received thymectomy or planned thymectomy during RCP within 6 months before randomization, or require chemotherapy and/or radiotherapy at any time;
5. Received rituximab treatment in the past 6 months before randomization;
6. Received tocilizumab or eculizumab treatment within 3 months before randomization;
7. Received IVIG or plasma exchange within 4 weeks before randomization;
8. Unresected thymoma.
9. History of other tumor diseases.

Where this trial is running

Beijing, Beijing Municipality and 5 other locations

• Beijing Tiantan Hospital, Capital Medical University — Beijing, Beijing Municipality, China (Not_yet_recruiting)
• Xiangya Hospital Central South University — Changsha, Hunan, China (Not_yet_recruiting)
• Tangdu Hospital, The Fourth Military Medical University — Xi'an, Shaanxi, China (Recruiting)
• Huashan Hospital — Shanghai, Shanghai Municipality, China (Not_yet_recruiting)
• West China Hospital of Sichuan University — Chengdu, Sichuan, China (Not_yet_recruiting)
• Tianjin medical university general hospital — Tianjin, Tianjin Municipality, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Ting Chang, MD,PHD

Conditions Myasthenia Gravis, Generalized; Generalized Myasthenia Gravis; Tocilizumab