Phase 3 Study to Assess Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants with gMG

JRCT ID: jRCT2021230001

Registered date:11/04/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Generalized Myasthenia Gravis
• Date of first enrollment: 01/06/2023
• Target sample size: 15
• Countries of recruitment: Canada,Japan,US,Japan,Argentina,Japan,Mexico,Japan,Brazil,Japan,Germany,Japan,Italy,Japan,Spain,Japan,UK,Japan,Hungary,Japan,Poland,Japan,Romania,Japan,Serbia,Japan,Georgia,Japan,South Korea,Japan
• Study type: Interventional

Intervention(s)

- Period 1: Induction Period
Period 1 (Week 0 to Week 12) is a randomized, 3-arm, parallel-group study designed to assess the safety and efficacy of batoclimab 340 mg subcutaneous (SC) once a week (QW) or 680 mg SC QW compared to PBO as Induction Therapy.

- Period 2: Maintenance Period
At completion of Period 1, eligible participants will enter Period 2 (Week 12 to Week 24) and receive batoclimab 340 mg SC QW, batoclimab 340 mg SC once every 2 weeks (Q2W), or PBO according to their treatment assignment in Period 1 and their change from Period 1 baseline in MG-ADL score (< 2 or >= 2-point improvement (decrease) from Period 1 baseline) at Weeks 10 and 12.

- Period 3: Long-term Extension (LTE) Period
Period 3 (Week 24 to Week 76) is a LTE assessing the safety and efficacy of batoclimab 340 mg SC QW and 340 mg SC Q2W through an additional 52 weeks of treatment.

Outcome(s)

• Primary Outcome: Change from Period 1 baseline in MG-ADL score to Week 12 for AChRAb+ participants (Timeframe: Period 1 baseline to Week 12)

Key inclusion & exclusion criteria

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

Include criteria

1. Age >= 18 years of age at the Screening Visit.
2. Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification Class II, III, or IVa at the Screening Visit.
3. Have a QMG score >= 11 at the Screening and Baseline Visits.
4. Have a MG-ADL score of >= 5 at the Screening and Baseline Visits.
5. Additional inclusion criteria are defined in the protocol.

Exclude criteria

1. Have experienced myasthenic crisis within 3 months of the Screening Visit.
2. Have had a thymectomy performed < 6 months prior to the Screening Visit or have a planned thymectomy during the study period.
3. Have any active or untreated malignant thymoma.
4. Have received any agent or therapy (exclusive of those identified within inclusion criteria) with immunosuppressive properties (e.g., stem cell therapy, chemotherapies, etc.) within the past year.
5. Have used anti-FcRN treatment within 3 months prior to the Screening Visit or have a documented history of non-response to prior anti-FcRN treatment.
6. Additional exclusion criteria are defined in the protocol.

Related Information

• Primary Sponsor: Yamazaki Kenji
• Secondary ID(s): 2021-000249-42,NCT05403541