A Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy - ACCESS-1

Regeneron Pharmaceuticals, Inc.0 个研究点目标入组 0 人开始时间: 待定最近更新: 2024年7月22日

试验速览

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•1.Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes as described in the protocol
•2.Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol
•3.LDH level greater than or equal to 2 into ULN at the screening visit
•4.Other protocol defined Inclusion Criteria apply

•1.Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening
•2.Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
•3.Body weight less than 40 kilograms at screening visit
•4.Planned use of any complement inhibitor therapy other than study drugs during the treatment period
•5.Not meeting meningococcal vaccination requirements for ravulizumab (Cohort A) or eculizumab (Cohort B) according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit
•6.Any contraindication for receiving Neisseria meningitidis vaccination
•7.Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab [Cohort A] or eculizumab [Cohort B] prescribing information, where available, or national guidelines/local practice or if necessary when vaccination is less than 2 weeks from study treatment initiation)
•8.Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
•9.Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
•10.Other protocol defined Exclusion Criteria apply

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