Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

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

Summary

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

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

Details

• Lead Sponsor: Alexion Pharmaceuticals
• Treatments: Eculizumab

Criteria

Inclusion Criteria:

• Must have required at least 4 transfusions in the past 12 months

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

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

• Platelet count > 100,000/mm3

• Patient taking erythropoietin must be on a stable dose for at least 26 weeks

• Patient taking immunosuppressants must be on a stable dose for at least 26 weeks

• Patient taking corticosteroids must be on a stable dose for at least 4 weeks

• Patient taking coumadin must be at a stable INR for at least 4 weeks

• Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks

• Willing and able to give written informed consent

• Must avoid conception

Exclusion Criteria:

• Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl

• Absolute neutrophil count <500/ul

• Active bacterial infection

• Hereditary complement deficiency

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

• Pregnant, breast-feeding, or intending to conceive

• History of meningococcal disease

• History of bone marrow transplantation