Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
- Conditions
- Hemoglobinuria, Paroxysmal
- Registration Number
- NCT00122304
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 85
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs) Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
- Secondary Outcome Measures
Name Time Method Hemolysis measured by the change of LDH from baseline; Quality of Life
Trial Locations
- Locations (48)
Norris Comprehensive Cancer Center
🇺🇸Los Angeles, California, United States
Stanford University Medical Center, Division of Hematology
🇺🇸Stanford, California, United States
Hartford Hospital, Cancer Clinical Research Office
🇺🇸Hartford, Connecticut, United States
Cleveland Clinic Florida, Dept. of Clinical Research
🇺🇸Weston, Florida, United States
Indiana University Cancer Pavilion
🇺🇸Indianapolis, Indiana, United States
Johns Hopkins University Medical Center
🇺🇸Baltimore, Maryland, United States
National Heart, Lung, and Blood Institute, National Institutes of Health
🇺🇸Bethesda, Maryland, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Mayo Clinic, Division of Hematology
🇺🇸Rochester, Minnesota, United States
Washington University Medical Center, Department of Internal Medicine/Division of Hematology
🇺🇸St. Louis, Missouri, United States
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