Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
2 other identifiers
interventional
85
13 countries
48
Brief Summary
The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Dec 2004
48 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 1, 2004
CompletedFirst Submitted
Initial submission to the registry
July 19, 2005
CompletedFirst Posted
Study publicly available on registry
July 22, 2005
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2006
CompletedFebruary 21, 2007
February 1, 2007
July 19, 2005
February 20, 2007
Conditions
Outcome Measures
Primary Outcomes (2)
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 Outcomes (2)
Hemolysis measured by the change of LDH from baseline;
Quality of Life
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (48)
Norris Comprehensive Cancer Center
Los Angeles, California, 90033, United States
Stanford University Medical Center, Division of Hematology
Stanford, California, 94305, United States
Hartford Hospital, Cancer Clinical Research Office
Hartford, Connecticut, 06102, United States
Cleveland Clinic Florida, Dept. of Clinical Research
Weston, Florida, 33331, United States
Indiana University Cancer Pavilion
Indianapolis, Indiana, 46202, United States
Johns Hopkins University Medical Center
Baltimore, Maryland, 21205, United States
National Heart, Lung, and Blood Institute, National Institutes of Health
Bethesda, Maryland, 20892, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Mayo Clinic, Division of Hematology
Rochester, Minnesota, 55905, United States
Washington University Medical Center, Department of Internal Medicine/Division of Hematology
St Louis, Missouri, 63110, United States
Cooper University Hospital, Cooper Cancer Institute
Voorhees Township, New Jersey, 08043, United States
Roswell Park Cancer Institute
Buffalo, New York, 14263, United States
NYU Clinical Cancer Center
New York, New York, 10016, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10017, United States
Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
Durham, North Carolina, 27710, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
OHSU Center for Hematologic Malignancies
Portland, Oregon, 97239, United States
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
Philadelphia, Pennsylvania, 19104, United States
University of Utah, Hematology-BMT Department
Salt Lake City, Utah, 84132, United States
Royal North Shore Hospital, Haematology Department
Saint Leonard, New South Wales, 2065, Australia
Princess Alexandra Hospital, Oncology Haematology Radiation Department
Woolloongabba, Queensland, 4102, Australia
The Queen Elizabeth Hospital, Haematology/Oncology Department
Woodville South, South Australia, 5011, Australia
The Royal Perth Hospital, Dept. of Haematology/Level 2
Perth, Western Australia, 6000, Australia
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
Parkville, 3050, Australia
Ucl St. Luc, Hematology Department
Brussels, 1200, Belgium
University of Alberta, Cross Cancer Institute
Edmonton, Alberta, T6G 2B7, Canada
London Regional Cancer Centre, Clinical Research Unit Room C3080
London, Ontario, N6A 4L6, Canada
Hopital Saint-Louis, Centre d'investigation Clinique
Paris, Cedex, 10 75475, France
Universitatsklinikum Essen, Zentrum fur Innere Medizin
Essen, D-45147, Germany
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
Hanover, D-30625, Germany
Universitatskliniken des Saarlandes, Innere Medizin 1
Homburg/Saar, D-66421, Germany
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm
Ulm, D-89081, Germany
St. James Hospital, Haematology Dept., Cancer Clinical Trial Office
Dublin, 8, Ireland
Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
Florence, 50139, Italy
Ospedale San Martino, Dept. of Hematology
Genova, I-16132, Italy
Ospedale Maggiore di Milano, Divisione di Ematologia
Milan, 35-20122, Italy
Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
Napoli, 5-80131, Italy
Ospedale San Bortolo, Divisione di Ematologia
Vicenza, 37-36100, Italy
UMC St. Radboud, Department of Hematology
Nijmegen, 6525 GA, Netherlands
Hospital Clinic i Provincial, Servicio de Hematologia
Barcelona, 08036, Spain
Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
Barcelona, 08916, Spain
Hospital De La Paz, Servicio de Hematologia
Madrid, 28046, Spain
Stockholm South Hospital, Division of Hematology
Stockholm, 118-83, Sweden
University Hospital, Dept. of Haematology
Uppsala, SE-751, Sweden
Kantonsspital Basel, Abteilung fuer Haematologie
Basel, CH-4031, Switzerland
Royal Cornwall Hospital
Truro, Cornwall, TR1 3LJ, United Kingdom
Leeds General Infirmary, D Floor Brotherton Wing
Leeds, LS1 3EX, United Kingdom
St. Georges Hospital, Department of Haematology
London, 17 OQT, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
July 19, 2005
First Posted
July 22, 2005
Study Start
December 1, 2004
Study Completion
November 1, 2006
Last Updated
February 21, 2007
Record last verified: 2007-02