Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
- Conditions
- Hemoglobinuria, Paroxysmal
- Registration Number
- NCT00122330
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 75
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (43)
University of California at Los Angeles
🇺🇸Los Angeles, California, United States
Washington University School of Medicine, Dept. of Internal Medicine/Division of Hematology
🇺🇸St. Louis, Missouri, United States
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
🇺🇸Philadelphia, Pennsylvania, United States
Ucl St. Luc, Hematology Department
🇧🇪Brussels, Belgium
Universitatsklinik Basel, Division of Hematology
🇨ðŸ‡Basel, CH, Switzerland
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
The Royal Perth Hosptial, Department of Haematology/Level 2
🇦🇺Perth, Western Australia, Australia
Stanford University Medical Center, Division of Hematology
🇺🇸Stanford, California, United States
Scripps Cancer Center
🇺🇸San Diego, California, United States
National Heart, Blood, and Lung Institute, National Institutes of Health
🇺🇸Bethesda, Maryland, United States
Johns Hopkins University Medical Center
🇺🇸Baltimore, Maryland, United States
Indiana University Cancer Pavilion, Div. of Hematology-Oncology, Hematological Malignancy Program/Immunology
🇺🇸Indianapolis, Indiana, United States
Ospedale San Bortolo, Divisione di Ematologia
🇮🇹Vicenza, Italy
Hospital De L'Hotel Dieu, Hematologie et Oncologie Medicale
🇫🇷Cedex, Paris, France
Memorial Sloan Kettering Cancer Center
🇺🇸New York, New York, United States
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
🇩🇪Hannover, Germany
City of Hope National Medical Center, Div. of Hematology & Bone Marrow Transplant
🇺🇸Duarte, California, United States
Saarland University Medical School, Internal Medicine 1
🇩🇪Homburg/Saar, Germany
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
🇦🇺Parkville, Victoria, Australia
University of Alberta, Cross Cancer Institute
🇨🇦Edmonton, Alberta, Canada
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionsmedizin des Universitatskinikums Ulm
🇩🇪Ulm, Germany
St. James Hospital, Haematology Department
🇮🇪Dublin, Ireland
Ospedale San Martino, Dept. of Hematology
🇮🇹Genova, Italy
Ospedale Maggiore di Milano, Divisione di Ematologia
🇮🇹Milano, Italy
Universitar degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
🇮🇹Napoli, Italy
UMC St. Radboud, Dept. of Hematology
🇳🇱Nijmegen, GA, Netherlands
Lund University Hospital, Department of Internal Medicine, Section of Hematology
🇸🇪Lund, Sweden
Umea University Hospital, Dept. of Internal Medicine 3, Section for Hematology
🇸🇪Umea, Sweden
Royal Cornwall Hospital, Haematology Dept.
🇬🇧Truro, Cornwall, United Kingdom
Hartford Hospital, Cancer Clinical Research Office
🇺🇸Hartford, Connecticut, United States
Cleveland Clinic, Dept. of Clinical Research
🇺🇸Weston, Florida, United States
NYU Clinical Cancer Center
🇺🇸New York, New York, United States
Duke University Health System, Division of Cell Therapy, Heme Malignancies Program
🇺🇸Durham, North Carolina, United States
Princess Alexandra Hospital, Oncology Haematology Radiation Department
🇦🇺Woolloongabba, Queensland, Australia
Hopital Saint-Louis, Centre G. Hayem-Secteur Bleu Porte 7, Laboratoire De Pathologie/Greffe de Moelle
🇫🇷Cedex, Paris, France
Universitatsklinikum Essen, Zentrum fur Innere Medizin
🇩🇪Essen, Germany
Universitatsklinik Greifswald, Innere Medizin C - Hamato-Onkologie
🇩🇪Greifswald, Germany
Queen Elizabeth Hospital, Dept. of Haematology
🇦🇺Woodville South, South Australia, Australia
Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
🇮🇹Firenze, Italy
Leeds General Infirmary, D Floor Brotherton Wing
🇬🇧Leeds, United Kingdom
Belfast City Hospital, Dept. of Haematology C Floor
🇬🇧Belfast, United Kingdom
St. George's Hospital, Department of Haematology
🇬🇧London, United Kingdom