Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.

The objective of this study was to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH. In addition, the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in Japanese patients were assessed and compared to that of non-Japanese patients who had participated earlier studies of eculizumab treatment in hemolytic PNH.

Inclusion Criteria:

• Japanese individuals at least 12 years of age
• Diagnosis of PNH > 6 months
• At least one transfusion in the past 2 years for anemia or anemia-related symptoms
• LDH level ≥ 1.5 x upper limit of normal within 12 weeks
• Presence of a glycosylphosphatidylinositol (GPI)-deficient red blood cell (RBC) clone (type III cells) by flow cytometry of ≥10%
• Negative serum pregnancy test for women of child-bearing potential

Exclusion Criteria:

• Platelet count < 30,000/µL
• Absolute neutrophil count ≤ 500/µL
• Known or suspected hereditary complement deficiency
• History of hematopoietic stem cell transplant
• History of meningococcal disease