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)
NYU Clinical Cancer Center
🇺🇸New York, New York, United States
Johns Hopkins University Medical Center
🇺🇸Baltimore, Maryland, United States
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
🇺🇸Philadelphia, Pennsylvania, United States
Princess Alexandra Hospital, Oncology Haematology Radiation Department
🇦🇺Woolloongabba, Queensland, Australia
National Heart, Lung, and Blood Institute, National Institutes of Health
🇺🇸Bethesda, Maryland, United States
Norris Comprehensive Cancer Center
🇺🇸Los Angeles, California, United States
Washington University Medical Center, Department of Internal Medicine/Division of Hematology
🇺🇸St. Louis, Missouri, United States
Ospedale San Martino, Dept. of Hematology
🇮🇹Genova, Italy
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
🇩🇪Hannover, Germany
Memorial Sloan Kettering Cancer Center
🇺🇸New York, New York, United States
Cleveland Clinic Florida, Dept. of Clinical Research
🇺🇸Weston, Florida, United States
Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
🇮🇹Firenze, Italy
University of Alberta, Cross Cancer Institute
🇨🇦Edmonton, Alberta, Canada
Universitatsklinikum Essen, Zentrum fur Innere Medizin
🇩🇪Essen, Germany
Hospital Clinic i Provincial, Servicio de Hematologia
🇪🇸Barcelona, Spain
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm
🇩🇪Ulm, Germany
University Hospital, Dept. of Haematology
🇸🇪Uppsala, Sweden
Hopital Saint-Louis, Centre d'investigation Clinique
🇫🇷Paris, Cedex, France
Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
🇪🇸Barcelona, Spain
Hospital De La Paz, Servicio de Hematologia
🇪🇸Madrid, Spain
UMC St. Radboud, Department of Hematology
🇳🇱Nijmegen, Netherlands
Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
🇮🇹Napoli, Italy
Ospedale San Bortolo, Divisione di Ematologia
🇮🇹Vicenza, Italy
Stockholm South Hospital, Division of Hematology
🇸🇪Stockholm, Sweden
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Stanford University Medical Center, Division of Hematology
🇺🇸Stanford, California, United States
Hartford Hospital, Cancer Clinical Research Office
🇺🇸Hartford, Connecticut, United States
Cooper University Hospital, Cooper Cancer Institute
🇺🇸Voorhees, New Jersey, United States
The Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
Roswell Park Cancer Institute
🇺🇸Buffalo, New York, United States
Royal North Shore Hospital, Haematology Department
🇦🇺St. Leonard, New South Wales, Australia
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
🇦🇺Parkville, Australia
St. Georges Hospital, Department of Haematology
🇬🇧London, United Kingdom
The Royal Perth Hospital, Dept. of Haematology/Level 2
🇦🇺Perth, Western Australia, Australia
Ucl St. Luc, Hematology Department
🇧🇪Brussels, Belgium
Kantonsspital Basel, Abteilung fuer Haematologie
🇨ðŸ‡Basel, Switzerland
Royal Cornwall Hospital
🇬🇧Truro, Cornwall, United Kingdom
St. James Hospital, Haematology Dept., Cancer Clinical Trial Office
🇮🇪Dublin, Ireland
Ospedale Maggiore di Milano, Divisione di Ematologia
🇮🇹Milano, Italy
Leeds General Infirmary, D Floor Brotherton Wing
🇬🇧Leeds, United Kingdom
London Regional Cancer Centre, Clinical Research Unit Room C3080
🇨🇦London, Ontario, Canada
Indiana University Cancer Pavilion
🇺🇸Indianapolis, Indiana, United States
Mayo Clinic, Division of Hematology
🇺🇸Rochester, Minnesota, United States
Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
🇺🇸Durham, North Carolina, United States
OHSU Center for Hematologic Malignancies
🇺🇸Portland, Oregon, United States
University of Utah, Hematology-BMT Department
🇺🇸Salt Lake City, Utah, United States
Universitatskliniken des Saarlandes, Innere Medizin 1
🇩🇪Homburg/Saar, Germany
The Queen Elizabeth Hospital, Haematology/Oncology Department
🇦🇺Woodville South, South Australia, Australia