Myasthenia Gravis, Generalized
Conditions
Keywords
Generalized Myasthenia Gravis, Tocilizumab
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.
Interventions
Participants will receive IV tocilizumab
Sponsors
Tang-Du Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in Quantitative Myasthenia Gravis (QMG) scores. | 16 weeks |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
| Proportion of subjects with both (1) ≥ 3-point improvement in QMG and (2) lasts ≥4 weeks | 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 |
| Change in Myasthenia Gravis Quality of Life-15, revised (MGQOL-15r) score. | 16 weeks |
Countries
China
Contacts
Primary ContactTing Chang, MD,PHD
Backup ContactZhe Ruan
Outcome results
None listed