Hemoglobinuria, Paroxysmal
Conditions
Brief summary
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.
Interventions
DRUGeculizumab
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
ECT
Masking
DOUBLE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Countries
Australia, Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Sweden, Switzerland, United Kingdom, United States
Outcome results
None listed