Batoclimab in MG
Trial question
What is the role of batoclimab in patients with generalized myasthenia gravis?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
67.0% female
33.0% male
N = 131
131 patients (88 female, 43 male).
Inclusion criteria: adult patients with generalized myasthenia gravis.
Key exclusion criteria: thymectomy within 3 months before screening; IVIG, plasma adsorption, or plasma exchange therapy within 4 weeks before screening; use of an immunosuppressive monoclonal antibody within 6 months before screening; HCV or HIV infection.
Interventions
N=67 batoclimab (each cycle of 6 weekly injections of batoclimab 680 mg, with 2 cycles maximum per patient).
N=64 placebo (each cycle of 6 weekly injections of placebo, with 2 cycles maximum per patient).
Primary outcome
Sustained improvement in Myasthenia Gravis Activities of Daily Living score in first cycle in antibody-positive patients
58.2%
31.3%
58.2 %
43.7 %
29.1 %
14.6 %
0.0 %
Batoclimab
Placebo
Significant
increase ▲
NNT = 3
Significant increase in sustained improvement in Myasthenia Gravis Activities of Daily Living score in the first cycle in antibody-positive patients (58.2% vs. 31.3%; OR 3.45, 95% CI 1.62 to 7.35).
Secondary outcomes
Significant increase in sustained Quantitative Myasthenia Gravis improvement (64.2% vs. 40.6%; OR 2.62, 95% CI 1.29 to 5.33).
Significant increase in minimal symptom expression (25.4% vs. 4.7%; OR 6.81, 95% CI 1.85 to 25.11).
No significant difference in early improvement in Myasthenia Gravis Activities of Daily Living score (32.8% vs. 34.4%; OR 0.95, 95% CI 0.44 to 2.05).
Safety outcomes
No significant difference in treatment-emergent adverse events.
Conclusion
In adult patients with generalized myasthenia gravis, batoclimab was superior to placebo with respect to sustained improvement in Myasthenia Gravis Activities of Daily Living score in the first cycle in antibody-positive patients.
Reference
Chong Yan, Yaoxian Yue, Yuzhou Guan et al. Batoclimab vs Placebo for Generalized Myasthenia Gravis: A Randomized Clinical Trial. JAMA Neurol. 2024 Mar 4;81(4):336-345. Online ahead of print.
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