Zilucoplan for severe gMG flares that require hospitalization.
Zilucoplan Treatment of Severe MG Exacerbations Leading to Hospitalization of Participants With Acetylcholine Receptor Antibody Positive gMG
This trial will try daily subcutaneous zilucoplan in adults with AChR-positive generalized myasthenia gravis who are hospitalized for severe flares to see if it quickly improves breathing and bulbar symptoms.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Ohio State University Academic / other |
| Drugs / interventions | Rituximab |
| Locations | 1 site (Columbus, Ohio) |
| Trial ID | NCT07215949 on ClinicalTrials.gov |
What this trial studies
This open-label, multicenter phase 3b trial will enroll about 15 adults with AChR antibody–positive generalized myasthenia gravis who are hospitalized for severe exacerbations. Participants will receive weight-based daily subcutaneous zilucoplan for 12 weeks, starting in the hospital and continuing as outpatients, with total participation around 18 weeks. Patients may choose standard-of-care plasma exchange or IVIG instead of the research treatment, and all participants will have regular assessments including QMG, MG-ADL, MGQoL15r, labs, and safety monitoring. Subjects must receive meningococcal vaccination prior to starting zilucoplan and will take prophylactic antibiotics until vaccination is complete.
Who should consider this trial
Good fit: Adults (≥18) with AChR antibody–positive generalized myasthenia gravis experiencing a severe exacerbation requiring hospitalization (MGFA II–IVb and MG‑ADL ≥6) who can be vaccinated and comply with study procedures are ideal candidates.
Not a fit: Patients with only ocular MG, who are AChR antibody–negative, who cannot be vaccinated or take prophylactic antibiotics, or who are already improving on standard IVIG/plasma exchange may not receive benefit from this approach.
Why it matters
Potential benefit: If successful, zilucoplan could offer a faster, less invasive way to relieve life‑threatening breathing and swallowing problems during severe MG exacerbations and may reduce hospital stay or need for plasma exchange/IVIG.
How similar studies have performed: Complement inhibitors including intravenous agents have shown benefit in AChR+ gMG and subcutaneous zilucoplan has shown efficacy in chronic gMG, but using zilucoplan specifically for acute hospitalized exacerbations is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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 be willing to use an acceptable method of birth control for the duration of the study and for 40 days after the last dose of study drug therapy. Acceptable methods of birth control include abstinence, oral contraceptives, the contraceptive patch, intra-uterine device, the contraceptive ring, and or barrier contraception such as condoms with spermicide. * Completed or updated meningococcal vaccination or initiated meningococcal vaccination with appropriate antibiotic prophylaxis according to current USPI and ACIP guidelines Exclusion Criteria: * History of meningococcal disease * Participants requiring intubation prior to study start. * Recent significant infections which could have caused exacerbation e.g. sepsis and wound infections * Pregnancy or lactating * Recent surgery (\<4 weeks). Minor procedures/surgeries allowed at the discretion of the site principal investigator * Current use or known failure of C5 inhibitors in the previous 3 months * Initiation of plasma exchange or IVIG in the past 4 weeks * Participation in concurrent clinical trial with a therapeutic medication * Rituximab use in the previous 9 months * Any clinically significant condition or illness, which, in the opinion of the PI, would pose a risk to the subject or might confound the study
Where this trial is running
Columbus, Ohio
- The Ohio State University — Columbus, Ohio, United States (Recruiting)
Study contacts
- Principal investigator: Miriam Freimer, MD — Ohio State University
- Study coordinator: Julie Agriesti, MACPR, CCRC
- Email: Julie.Agriesti@osumc.edu
- Phone: 614-293-4098
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.