Myasthenia Gravis Inebilizumab Trial

JRCT ID: jRCT2021210003

Registered date:21/04/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: AChR-Ab+ or MuSK-Ab+ generalized Myasthenia Gravis
• Date of first enrollment: 18/08/2021
• Target sample size: 16
• Countries of recruitment: USA,Japan,Ukraine,Japan,China,Japan,Argentina,Japan,Brazil,Japan,Canada,Japan,France,Japan,Denmark,Japan,Germany,Japan,Israel,Japan,India,Japan,Italy,Japan,Poland,Japan,Spain,Japan,Belarus,Japan,Russia,Japan,Taiwan,Japan,Turkey,Japan,South Korea,Japan
• Study type: Interventional

Intervention(s)

(RCP: Randomized Controlled Period)
Treatment group 1: AChR-Ab positive population (active) - inebilizumab 300 mg administered intravenously (IV) on Days 1, 15, and 183 of the RCP
Treatment group 2: AChR-Ab positive population (placebo) - IV placebo on Days 1, 15, and 183 of the RCP
Treatment group 3: MuSK-Ab positive population (active) - inebilizumab 300 mg IV on Days 1 and 15 of the 26-week RCP
Treatment group 4: MuSK-Ab positive population (placebo) - IV placebo on Days 1 and 15 of the 26-week RCP

Outcome(s)

• Primary Outcome: Primary endpoint: Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at end of the RCP (Week 52 for AChR-Ab+ population and Week 26 for MuSK-Ab+ population).

Secondary Outcome

Key secondary endpoints:
1. Change in Quantitative Myasthenia Gravis (QMG) scores at the end of the RCP.
2. Proportion of subjects with both (1) greater than or equal to 3-point improvement in MG-ADL at end of RCP and (2) For AChR-Ab+ Population: did not initiate rescue therapy between Week 44 and Week 52 during the RCP; for MuSK-Ab+ population: did not initiate rescue therapy between Week 22 and Week 26 during the RCP.
3. Change in MG-ADL at Week 26 in the AChR-Ab positive population. Additional secondary endpoints:
4. Time to first exacerbation.
5. Change in Myasthenia Gravis Composite (MGC) score at the end of the RCP.
6. Change in Myasthenia Gravis Quality of Life-15, revised (MGQOL-15r) score at the end of the RCP.
7. Change in Patient Global Impression of Change score at the end of the RCP.
8. The safety and tolerability of inebilizumab as measured by the incidence of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and treatment-emergent serious adverse events (TESAEs). Laboratory measurements will also be evaluated.
9. PK profile of inebilizumab.
10. Anti-drug antibody (ADA) status and titer.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Diagnosis of MG with anti-AChR or anti-MuSK antibody.
2. MGFA Clinical Classification Class II, III, or IV.
3. MG-ADL score at the time of screening and randomization between 6 and 10 with > 50% of this score attributed to non-ocular items, or an MG-ADL score 11 or greater.
4. QMG score of 11 or greater.
5. Subjects must be on:
a. Corticosteroids only, with no dose increase within 4 weeks prior to randomization, or
b. One allowed non-steroidal IST, with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization, or
c. Combination of (1) corticosteroids with no dose increase within 4 weeks prior to randomization and (2) one allowed non-steroidal IST with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization.
Allowed ISTs, alone or in combination with corticosteroids, are azathioprine, mycophenolate mofetil, and mycophenolic acid.
Tacrolimus is allowed in Japan only, at a dose of < 3 mg/day, with continued use for at least 6 months prior
to randomization and no dose increase within 4 months prior to randomization.

Exclude criteria

1. Receipt within the 4 weeks prior to Day 1:
a. Cyclosporine (except eye drops)
b. Tacrolimus (except topical) (tacrolimus < 3 mg/day is allowed in Japan only)
c. Methotrexate
2. Current use of:
a. Prednisone > 40 mg/day or > 80 mg over a 2-day period (or equivalent dose of other corticosteroids)

Related Information

• Primary Sponsor: Cardona Ritsuko
• Secondary ID(s): NCT04524273,2020-000949-14