{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Howard JF"],"funding":["NCATS NIH HHS","NINDS NIH HHS"],"pagination":["582-592"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC7042797"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["77(5)"],"pubmed_abstract":["<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. Fifty"],"journal":["JAMA neurology"],"pubmed_title":["Clinical 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."],"pmcid":["PMC7042797"],"funding_grant_id":["U24 NS107128","UL1 TR001863"],"pubmed_authors":["Gibson S","Chopra M","Benatar M","Patterson K","Pillai R","Duda PW","Aly R","Nye J","Barohn R","Kohli A","Patrick K","MacDougall J","Traub RE","Sharma A","Elsheikh B","Hinton JL","Olapo T","Farmakidis C","Khatri B","Lewis-Collins TD","Kaminski HJ","Wolfe GI","Siddiqi Z","Lindman E","Berger A","Benatar MG","Hoarty M","Freimer ML","Bromberg M","Vu TH","Sershon LM","Mozaffar T","Ho D","Bayat E","Holzberg S","Matisak P","Sachdev A","Lisak RP","Silvestri N","Pasnoor M","Howard JF","Zakalik K","Harvey B","Dimachkie M","Colgan S","Henegar R","Pontius A","Mathew V","Pulley M","Sullivan P","Abu-Rub M","MacDougall JE","Liu T","Bernitsas E","Jia K","Bratton J","Fetter M","David W","Shabbir Y","Bertorini T","Freimer M","Genge A","Hewitt W","Farzaneh-Far R","Janecki T","Small G","Lange D","Malik R","Guidon A","Nicolle M","Roy B","Sivak M","Nowak RJ","Zilucoplan MG Study Group","Lam L","Barnett D","Vu T","Habib AA","Khan S"],"additional_accession":[]},"is_claimable":false,"name":"Clinical 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.","description":"<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. Fifty","dates":{"release":"2020-01-01T00:00:00Z","publication":"2020 May","modification":"2026-05-01T16:53:14.89Z","creation":"2021-02-19T23:32:37Z"},"accession":"S-EPMC7042797","cross_references":{"pubmed":["32065623"],"doi":["10.1001/jamaneurol.2019.5125"]}}