A RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-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

Regeneron Pharmaceuticals, Inc.0 个研究点目标入组 124 人开始时间: 2022年3月11日最近更新: 2022年5月16日

适应症

试验速览

暂无简介。

•1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes described in the protocol
•2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms described in the protocol 3. LDH level =2 × ULN at the screening visit
•Note: Other protocol-defined Inclusion Criteria apply
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 108
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 16

•1. Prior treatment with a complement inhibitor within 6 months prior to screening visit, unless patient was treated with eculizumab or ravulizumab and has documented C5 variant R885H/C in which case there is no exclusion of such patients
•2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
•3. Body weight <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 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 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
•Note: Other protocol-defined Exclusion Criteria apply

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