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A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Cold
Agglutinin Disease (CAD)

Primary objective:- To demonstrate the efficacy of twice-weekly s.c. 1080-mg infusions of pegcetacoplan compared with that of placebo in patients with CAD.Secondary objectives:- To demonstrate the effect of pegcetacoplan on the number of PRBC…

AN OPEN-LABEL, RANDOMIZED, CONTROLLED, PHASE 2 STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PEGCETACOPLAN IN THE TREATMENT OF POST-TRANSPLANT RECURRENCE OF C3G OR IC*MPGN

This study has been transitioned to CTIS with ID 2024-511544-36-00 check the CTIS register for the current data. Primary:To evaluate the efficacy of pegcetacoplan in improving the underlying pathophysiology of complement 3 glomerulopathy (C3G)/…

A Phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS)

The primary objective of this study is to assess the efficacy of twice per week subcutaneous (SC) doses of pegcetacoplan 1080 mg compared toplacebo in subjects with sporadic ALS as measured by the Combined Assessment of Function and Survival (CAFS)…

An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria

The objectives of the study are to assess safety and tolerability, pharmacokinetics (PK), and biological activity (including efficacy and pharmacodynamics [PD]) of multiple subcutaneous doses of pegcetacoplan in pediatric patients with paroxysmal…

A phase 3, randomized, placebo-controlled, double-blinded, multicenter study to evaluate the efficacy and safety of pegcetacoplan in patients with C3 glomerulopathy or immune-complex membranoproliferative glomerulonephritis

This study has been transitioned to CTIS with ID 2024-514130-20-00 check the CTIS register for the current data. The primary objective of this study is to assess the efficacy of twice-weekly SC doses of pegcetacoplan compared with that of placebo in…