A Phase 3 interventional study of Zilucoplan® in Generalized Myasthenia Gravis (gMG), sponsored by Miriam Freimer. Recruiting at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-28.
Sponsored by Miriam Freimer · Phase 3, Interventional, and Treatment
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). Assessments will include medical history, physical exam, vital signs and bloodwork. MG assessments will also be conducted including the QMG and MG-ADL and patient reported outcome measures like the Myasthenia Gravis Quality of Life 15 item Revised (MGQoL15r) scale. Treatment will start in the hospital and once the patient is discharged, treatment will continue as an outpatient. Current medications and adverse events will also be reviewed and documented. Patients who undergo treatment with zilucoplan will be vaccinated against meningitis prior to starting the study drug and will continue with the recommended set of vaccines for 6 months. Antibiotics will be taken concurrently until patients are fully vaccinated.
The study aims to find a less invasive method to treat MG patients who are experiencing an exacerbation. Utilization of a subcutaneous medication with relatively rapid onset of action may provide a viable alternative to the current therapeutic approaches. If rapid complement inhibition were to prove efficacious in the treatment of acute exacerbations of gMG, this therapy would potentially obviate the need for transfer to tertiary care centers, avoid the potentially hazardous placement of a large bore central venous catheter for plasmapheresis, and potentially reduce hospital length of stay.
324 studies on the registry are indexed under Myasthenia Gravis; 146 are open to participants now.
This study's planned enrollment of 15 is below the median of 44 across 212 interventional studies indexed under Myasthenia Gravis.
Browse Myasthenia Gravis studies →This is the only study on the registry with Miriam Freimer as lead sponsor.
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Exclusion Criteria:
Subcutaneous zilucoplan
Drug: Zilucoplan®
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.
Change from baseline to Week 2 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score
The MG-ADL is an assessment that measures MG symptom severity. There are 8 items measured on a scale of 0-3 with 0 being the least severe. The total of the 8 items represents the MG-ADL score. The score can range from 0 (least severe) to 24 (most severe).
Time frame: From baseline to Week 2
Change from baseline to week 12 in Myasthenia Gravis - Activities of Daily Living (MG-ADL) score
The MG-ADL is an assessment that measures MG symptom severity. There are 8 items measured on a scale of 0-3 with 0 being the least severe. The total of the 8 items represents the MG-ADL score. The score can range from 0 (least severe) to 24 (most severe).
Time frame: From baseline to week 12
Plan to share: No
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