Zilucoplan for Severe gMG Exacerbations

Korábbi kezelés

• Participants requiring intubation prior to study start.
• 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
• Participation in concurrent clinical trial with a therapeutic medication
• Rituximab use in the previous 9 months

Más betegség

• History of meningococcal disease
• Recent significant infections which could have caused exacerbation e.g. sepsis and wound infections
• 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

Egyéb

• Pregnancy or lactating
• Initiation of plasma exchange or IVIG in the past 4 weeks

• 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