Zilucoplan for severe gMG flares that require hospitalization.

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• The Ohio State University
  Columbus, Ohio, United States Recruiting
• University of South Florida/Tampa General Hospital
  Tampa, Florida, United States Not yet recruiting
• University of Kansas Medical Center
  Kansas City, Kansas, United States Not yet 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

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.