Status:

NOT_YET_RECRUITING

Zilucoplan for Severe gMG Exacerbations

Lead Sponsor:

Miriam Freimer

Collaborating Sponsors:

UCB Pharma

Conditions:

Generalized Myasthenia Gravis (gMG)

Eligibility:

All Genders

18+ years

Phase:

PHASE3

Brief 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 inj...

Detailed Description

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....

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

December 1 2025

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

April 1 2028

Estimated Enrollment :

15 Patients enrolled

Trial Details

Trial ID

NCT07215949

Start Date

December 1 2025

End Date

April 1 2028

Last Update

October 14 2025

Active Locations (1)

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1

The Ohio State University

Columbus, Ohio, United States, 43210