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Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study

Phase 3
18 Years
Not Enrolling
Hemoglobinuria, Paroxysmal

Thank you

Trial Information

Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study

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

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)


United States: Food and Drug Administration

Study ID:




Start Date:

December 2004

Completion Date:

November 2006

Related Keywords:

  • Hemoglobinuria, Paroxysmal
  • Hemoglobinuria
  • Hemoglobinuria, Paroxysmal



Memorial Sloan Kettering Cancer Center New York, New York  10021
Roswell Park Cancer Institute Buffalo, New York  14263
Massachusetts General Hospital Boston, Massachusetts  02114-2617
The Cleveland Clinic Foundation Cleveland, Ohio  
Indiana University Cancer Pavilion Indianapolis, Indiana  46202
Stanford University Medical Center, Division of Hematology Stanford, California  94305-5112
Norris Comprehensive Cancer Center Los Angeles, California  90033
Hartford Hospital, Cancer Clinical Research Office Hartford, Connecticut  06102
Cleveland Clinic Florida, Dept. of Clinical Research Weston, Florida  33331
Johns Hopkins University Medical Center Baltimore, Maryland  21205
National Heart, Lung, and Blood Institute, National Institutes of Health Bethesda, Maryland  20892
Mayo Clinic, Division of Hematology Rochester, Minnesota  55905
Washington University Medical Center, Department of Internal Medicine/Division of Hematology St. Louis, Missouri  63110
Cooper University Hospital, Cooper Cancer Institute Voorhees, New Jersey  08043
NYU Clinical Cancer Center New York, New York  10016
Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program Durham, North Carolina  27710
OHSU Center for Hematologic Malignancies Portland, Oregon  97239
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program Philadelphia, Pennsylvania  19104
University of Utah, Hematology-BMT Department Salt Lake City, Utah  84132