Efficacy and Safety of Tocilizumab in the Treatment of Generalized Myasthenia Gravis (tMG)

Tang-Du Hospital

Status and phase: Enrolling; Phase 2

Conditions: Myasthenia Gravis, Generalized

Treatments: Drug: Tocilizumab Injectable Product

Study type: Interventional

Funder types: Other

Identifiers

NCT05067348

V4.0, 20220410

Details and patient eligibility

About

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

Full 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.

Enrollment: 64 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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64 participants in 2 patient groups, including a placebo group

Tocilizumab

Experimental group

Description:

Participants will receive tocilizumab administered intravenously (IV) on weeks 1,5,9 and 13 of the randomized controlled period.

Treatment:

Drug: Tocilizumab Injectable Product

Placebo

Placebo Comparator group

Description:

Participants will receive placebo administered intravenously (IV) on weeks 1,5,9 and 13 of the randomized controlled period.

Treatment:

Drug: Tocilizumab Injectable Product