biostudies-literatureHoward JFNCATS NIH HHSNINDS NIH HHS582-592https://www.ebi.ac.uk/biostudies/studies/S-EPMC7042797biostudies-literatureUnknown77(5)<h4>Importance</h4>Many patients with generalized myasthenia gravis (gMG) have substantial clinical disability, persistent disease burden, and adverse effects attributable to chronic immunosuppression. Therefore, there is a significant need for targeted, well-tolerated therapies with the potential to improve disease control and enhance quality of life.<h4>Objective</h4>To evaluate the clinical effects of zilucoplan, a subcutaneously (SC) self-administered macrocyclic peptide inhibitor of complement component 5, in a broad population of patients with moderate to severe gMG.<h4>Design, setting, and participants</h4>This randomized, double-blind, placebo-controlled phase 2 clinical trial at 25 study sites across North America recruited participants between December 2017 and August 2018. FiftyJAMA neurologyClinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial.PMC7042797U24 NS107128UL1 TR001863Gibson SChopra MBenatar MPatterson KPillai RDuda PWAly RNye JBarohn RKohli APatrick KMacDougall JTraub RESharma AElsheikh BHinton JLOlapo TFarmakidis CKhatri BLewis-Collins TDKaminski HJWolfe GISiddiqi ZLindman EBerger ABenatar MGHoarty MFreimer MLBromberg MVu THSershon LMMozaffar THo DBayat EHolzberg SMatisak PSachdev ALisak RPSilvestri NPasnoor MHoward JFZakalik KHarvey BDimachkie MColgan SHenegar RPontius AMathew VPulley MSullivan PAbu-Rub MMacDougall JELiu TBernitsas EJia KBratton JFetter MDavid WShabbir YBertorini TFreimer MGenge AHewitt WFarzaneh-Far RJanecki TSmall GLange DMalik RGuidon ANicolle MRoy BSivak MNowak RJZilucoplan MG Study GroupLam LBarnett DVu THabib AAKhan SfalseClinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial.<h4>Importance</h4>Many patients with generalized myasthenia gravis (gMG) have substantial clinical disability, persistent disease burden, and adverse effects attributable to chronic immunosuppression. Therefore, there is a significant need for targeted, well-tolerated therapies with the potential to improve disease control and enhance quality of life.<h4>Objective</h4>To evaluate the clinical effects of zilucoplan, a subcutaneously (SC) self-administered macrocyclic peptide inhibitor of complement component 5, in a broad population of patients with moderate to severe gMG.<h4>Design, setting, and participants</h4>This randomized, double-blind, placebo-controlled phase 2 clinical trial at 25 study sites across North America recruited participants between December 2017 and August 2018. Fifty2020-01-01T00:00:00Z2020 May2026-05-01T16:53:14.89Z2021-02-19T23:32:37ZS-EPMC70427973206562310.1001/jamaneurol.2019.5125