Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab

• Status: Completed
• Trial end date: 2006-11-01
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals
• Treatments: Eculizumab

Criteria

Inclusion Criteria:

• PNH > 6 months

• Type III PNH red blood cell (RBC) clone by flow cytometry >10%

• At least one transfusion in the past 2 years but no more than 3 transfusions in the past 12 months; or personal beliefs that preclude the use of transfusion with severe hemolytic PNH

• Lactate dehydrogenase (LDH) >1.5 x upper limit of normal

• Must avoid conception

• Willing and able to give written informed consent

Exclusion Criteria:

• Platelet count of <30,000/mm3

• Absolute neutrophil count <500/ul

• Active bacterial infection

• Hereditary complement deficiency

• History of bone marrow transplantation

• Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days

• Pregnant, breast-feeding, or intending to conceive

• History of meningococcal disease