The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH
Official Title
Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
Conditions
Hemoglobinuria, Paroxysmal
Study Type
Interventional
Study Design
Educational/Counseling/Training, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
Further Details
Study Start
October 2004; Study completion: January 2006
Eligibility & Criteria
- Ages Eligible for Study: 18 Years and above
- Genders Eligible for Study: Both
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
Total Enrolment
75
Contact Details
- Princess Alexandra Hospital, Oncology Haematology Radiation Department, Woolloongabba, Queensland, 4102, Australia
- Queen Elizabeth Hospital, Dept. of Haematology, Woodville South, South Australia, 5011, Australia
- Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology, Parkville, Victoria, 3050, Australia
- The Royal Perth Hosptial, Department of Haematology/Level 2, Perth, Western Australia, 6000, Australia
Results published in The New England Journal of Medicine
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