Zilucoplan (Zilbrysq®) Disease area studied Abbreviated title Phase Study ID Registry database Study results Plain language summary Publication (if available) Generalized Myasthenia Gravis A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis Phase 2/3 MG0014 Ongoing NCT060559592022-502072-23-00 Kostera-Pruszczyk A.et al. Muscle and Nerve 2023; 68(4):667, AANEM (American Association of Neuromuscular & Electrodiagnostic Medicine), MGFA session, November 1-4, 2023; Phoenix, USA Generalized Myasthenia Gravis An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors Phase 3 MG0017 Completed NCT05514873 Freimer M., et al. Neurology 2024; 102(17 Suppl.1) 76th Annual Meeting of American Academy of Neurology (AAN), April 13-18, 2024; Denver, USA Paroxysmal Nocturnal Hemoglobinuria Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Phase 2 RA101495-01.201 Completed NCT030785822016-003522-16 LINKLINK Kulasekararaj A.G.et al. Haematologica 2024, 109(3):929-935 Paroxysmal Nocturnal Hemoglobinuria Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study Phase 2 RA101495-01.202 Terminated NCT032252872016-003523-34 Paroxysmal Nocturnal Hemoglobinuria Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab Phase 2 RA101495-01.203 Completed NCT03030183 LINK Kulasekararaj A.G.et al. Haematologica 2024, 109(3):929-935 Generalized Myasthenia Gravis Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis Phase 2 RA101495-02.201 Completed NCT03315130 LINK Howard J.F., JAMA neurology 2020; 77(5):582–92 Immune-Mediated Necrotizing Myopathy Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy Phase 2 RA101495-02.202 Terminated NCT040256322019-001497-29 LINKLINK Mammen A.L. et al. Lancet Rheumatology 2023; 5(2):e67-e76 Generalized Myasthenia Gravis Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE) Phase 3 RA101495-02.301 Completed NCT041152932019-001564-30 PDF Howard J.F. Lancet Neurol 2023; 22(5):395–406 Generalized Myasthenia Gravis Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis (RAISE-XT) Phase 3 RA101495-02.302 Ongoing NCT042258712024-512399-37-00 Freimer M. et al. Neurology 2023; 100(17 suppl.2), 75th Annual Meeting of American Academy of Neurology (AAN), April 22-27, 2023; Boston, USA Weiss M.D. et al. Journal of neurology 2024, 271(5): 2758-2767 Generalized Myasthenia Gravis An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis (ziMyG+) Phase 3 MG0015 Ongoing NCT064353122022-502073-42-00 Generalized Myasthenia Gravis A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis Phase 3 DV0013 Ongoing NCT064713612023-508287-30-00
