Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 3, Single-arm, Open-label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.

Inclusion Criteria:

• Adult C5 inhibitor naive PNH patients (age>=18), which is confirmed by flow cytometry evaluation.
• Must be vaccinated againast N meningitidis.

Exclusion Criteria

• Meningitidis infection or unresolved meningococcal disease
• History of bone marrow transplantation
• Other significant systemic diseases that might have impact on efficacy and safety assessment