NCT07215949 · Miriam Freimer
Zilucoplan for Severe gMG Exacerbations
What this study is about
This is an where both patients and doctors know the treatment given, conducted at multiple hospitals, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive injected under the skin zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks.
View original scientific description
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.
Interventions
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.
Primary outcome measures
Change from baseline to Week 2 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score
Time frame: From baseline to Week 2
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).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Columbus, Ohio
Collaborators
UCB Pharma
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Feb 23, 2026 · Source of record for eligibility and locations