Efficacy and Safety of Tocilizumab in the Treatment of Generalized Myasthenia Gravis

Phase 2

Recruiting

• Conditions: Myasthenia Gravis, Generalized
• Interventions: Drug: Tocilizumab Injectable Product

• Registration Number: NCT05067348
• Lead Sponsor: Tang-Du Hospital

Brief Summary

Randomized, double-blind, placebo-controlled, parallel-group study with optional open-label extension.

Detailed Description

This study is a randomized, double-blind, placebo-controlled study, to be conducted at 6 study sites. Approximately 64 subjects will be enrolled. Patients with MG who are positive for anti-AChR antibodies will be enrolled. Patients who do not have anti-AChR or anti-MuSK antibodies will not be enrolled. Patients with MGFA classification II, III, or IV disease, MG-ADL score ≥ 5, QMG score ≥ 11, and use of a corticosteroid and/or non-steroidal immunosuppressant will be included in the study.

All subjects who complete the randomized controlled period will have the option to enroll in a 1-year open-label period.

Study Timeline

• Study First Submitted: 2021-09-23
• Study First Submitted QC: 2021-09-23
• Study First Posted: 2021-10-05
• Study Start: 2022-07-21
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-28
• Last Update Posted: 2025-05-01
• Primary Completion: 2025-12-31
• Study Completion: 2026-03-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 64

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Tocilizumab	Tocilizumab Injectable Product	Participants will receive tocilizumab administered intravenously (IV) on weeks 1,5,9 and 13 of the randomized controlled period.
Placebo	Tocilizumab Injectable Product	Participants will receive placebo administered intravenously (IV) on weeks 1,5,9 and 13 of the randomized controlled period.

Primary Outcome Measures

Name	Time	Method
Change in Quantitative Myasthenia Gravis (QMG) scores.	16 weeks

Secondary Outcome Measures

Name	Time	Method
Change in Myasthenia Gravis Quality of Life-15, revised (MGQOL-15r) score.	16 weeks
Change in Myasthenia Gravis Impairment Index (MGII) score.	16 weeks
Number of participants with treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and treatment-emergent serious adverse events (TESAEs) during the randomized controlled period and open-label period.	16 weeks
Proportion of subjects with both (1) ≥ 3-point improvement in QMG and (2) lasts ≥4 weeks	16 weeks
Proportion of subjects with both (1) ≥ 2-point improvement in MG-ADL and (2) lasts ≥4 weeks	16 weeks
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Profile score.	16 weeks
Change in Myasthenia Gravis Composite (MGC) score	16 weeks

Trial Locations

Locations (6)

Tangdu Hospital, The Fourth Military Medical University; 🇨🇳 Xi'an, Shaanxi, China

Beijing Tiantan Hospital, Capital Medical University; 🇨🇳 Beijing, Beijing, China

Xiangya Hospital Central South University; 🇨🇳 Changsha, Hunan, China

Huashan Hospital; 🇨🇳 Shanghai, Shanghai, China

Tianjin medical university general hospital; 🇨🇳 Tianjin, Tianjin, China

West China Hospital of Sichuan University; 🇨🇳 Chengdu, Sichuan, China