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

Paroxysmal nocturnal haemoglobinuria (PNH)

Cohort A: Percent change in lactate dehydrogenase (LDH), Cohort B: Adequate control of hemolysis (defined as LDH ≤1.5 × ULN) at each visit, Cohort B: Transfusion avoidance (not requiring a red blood cell (RBC) transfusion per the protocol)

Maintenance of adequate control of hemolysis (defined as LDH ≤1.5 × ULN) (Cohort A and B), Breakthrough hemolysis (defined as LDH ≥2 × ULN per the protocol) (Cohort A and B), Adequate control of hemolysis (defined as LDH ≤1.5 × ULN) (Cohort A), Hemoglobin stabilization (defined as patients who do not receive an RBC transfusion and have no decrease in hemoglobin level per the protocol) (Cohort A and B), Normalization of LDH (defined as LDH ≤1.0 × ULN per the protocol) (Cohort A and B), Transfusion Avoidance (defined as Not requiring an RBC transfusion as per protocol algorithm based on post-baseline hemoglobin values) (Cohort A), Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT)-Fatigue Scale (Cohort A and B), Change in physical function (PF) scores on the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30) (Cohort A and B), Change in global health status (GHS)/QoL scale score on the EORTC-QLC- C30 (Cohort A and B), Percent change in LDH (Cohort B), Rate of RBC transfused per protocol algorithm (Cohort A and B), Number of units of RBC transfused per protocol algorithm (Cohort A and B), Time to first LDH ≤1.5 × ULN (Cohort A and B), Time to first LDH ≤1.0 × ULN (Cohort A and B), Percentage of days with LDH ≤1.5 × ULN (Cohort A and B), Change in hemoglobin levels (Cohort A and B), Incidence and severity of treatment emergent serious adverse events (SAEs) (Cohort A and B), Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest (Cohort A and B), Incidence and severity of TEAEs leading to treatment discontinuation (Cohort A and B), Change in total CH50 (Cohort A and B), Percent change in total CH50 (Cohort A and B), Concentration of total C5 in plasma (Cohort A and B), Concentrations of total pozelimab in serum (Cohort A and B), Concentrations of cemdisiran in plasma (Cohort A and B), Concentrations of total ravulizumab (Cohort A) and total eculizumab (Cohort B) in serum, Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab (Cohort A and B), Incidence of treatment emergent ADAs to cemdisiran (Cohort A and B)

No related papers found yet.

Greece, Hungary, Italy, Poland, Romania, Spain