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

Paroxysmal Nocturnal Hemoglobinuria

Pharmacokinetics: Pegcetacoplan concentrations and PK parameters over the course of the 16-week treatment period, Efficacy and Pharmacodynamics: Change from baseline to Week 16 in Hb level, Efficacy and Pharmacodynamics: Change from baseline to Week 16 in LDH level, Efficacy and Pharmacodynamics: Change from baseline to Week 16 in ARC, Safety: Incidence and severity of TEAEs over the course of the 16-week treatment period, including bacterial infections

Efficacy and Pharmacodynamics: Change from baseline to Week 16 and Week 52 in: − Complement levels (eg, total complement hemolytic activity [CH50], alternative complement pathway hemolytic activity [AH50], and C3 level) − C3 deposition on RBCs − Clonal distribution of PNH RBCs, Efficacy and Pharmacodynamics: Number of transfusions, number of packed RBC units, and total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan, Efficacy and Pharmacodynamics: Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan, Efficacy and Pharmacodynamics: Change from baseline to Week 52, and from Week 16 to Week 52, in hemoglobin, LDH, and ARC, Efficacy and Pharmacodynamics: Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life assessments (FACIT-Fatigue and PedsQL General Well-Being Scale), Safety: Incidence of thromboembolic events (major adverse vascular events [MAVE]) over the course of the 16-week treatment period and over 52 weeks of treatment with pegcetacoplan

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Netherlands, Spain