Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
Phase 3
18 Years
N/A
18 Years
N/A
Not Enrolling
Both
Hemoglobinuria, Paroxysmal
Both
Hemoglobinuria, Paroxysmal
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:
Interventional
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)
Authority:
United States: Food and Drug Administration
Study ID:
SHEPHERD
NCT ID:
NCT00122304
Start Date:
December 2004
Completion Date:
November 2006
Related Keywords:
- Hemoglobinuria, Paroxysmal
- Hemoglobinuria
- Hemoglobinuria, Paroxysmal
Name | Location |
|---|---|
| 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 |
