Miriam Freimer
This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks.
The primary objective of the study is to evaluate the efficacy of subcutaneous zilucoplan in participants with AChR antibody positive gMG who experience severe exacerbations requiring hospitalization. A total of 15 patients will be enrolled in the study with treatment lasting 12 weeks. Dosing will be weight based. Patients will be presented with the option of undergoing standard of care plasma exchange or IVIG or to participate in the research study to determine if the use of zilucoplan would rapidly alleviate the most severe symptoms of gMG (respiratory dysfunction and/or bulbar dysfunction)…
Inclusion Criteria: * Patient determined to have severe MG exacerbation (e.g. bulbar and/or respiratory symptoms requiring hospitalization, neck extension weakness) * MGFA class II - IVb * Male or female aged ≥18 * MG-ADL ≥6 in non-ocular domains * Serology - AChR antibody positive (or historically available data) * If female of child-bearing potential (i.e., not surgically sterile or post-menopausal defined as age \> 51 years without menses for ≥ 2 years), negative serum pregnancy test at screening * Women of child-bearing potential or men with sexual partners of childbearing potential must…
Subcutaneous injections of zilucoplan will be administered daily. The dose is dependent on the body weight of the patient. Each patient will receive a kit with prefilled syringes.
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