RecruitingPhase 3NCT07215949

Zilucoplan for Severe gMG Exacerbations

Zilucoplan Treatment of Severe MG Exacerbations Leading to Hospitalization of Participants With Acetylcholine Receptor Antibody Positive gMG


Sponsor

Miriam Freimer

Enrollment

15 participants

Start Date

Jan 20, 2026

Study Type

INTERVENTIONAL

Conditions

Summary

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.


Eligibility

Min Age: 18 Years

Inclusion Criteria8

  • 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 Criteria10

  • 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

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Interventions

DRUGZilucoplan®

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.


Locations(1)

The Ohio State University

Columbus, Ohio, United States

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NCT07215949


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