Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
1 other identifier
interventional
75
12 countries
43
Brief Summary
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with 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 Oct 2004
Shorter than P25 for phase_3
43 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
October 1, 2004
CompletedFirst Submitted
Initial submission to the registry
July 18, 2005
CompletedFirst Posted
Study publicly available on registry
July 22, 2005
CompletedStudy Completion
Last participant's last visit for all outcomes
January 1, 2006
CompletedDecember 4, 2006
November 1, 2006
July 18, 2005
November 30, 2006
Conditions
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (43)
City of Hope National Medical Center, Div. of Hematology & Bone Marrow Transplant
Duarte, California, 91010-3000, United States
University of California at Los Angeles
Los Angeles, California, 90024, United States
Scripps Cancer Center
San Diego, California, 92121, United States
Stanford University Medical Center, Division of Hematology
Stanford, California, 94305-5821, United States
Hartford Hospital, Cancer Clinical Research Office
Hartford, Connecticut, 06102, United States
Cleveland Clinic, Dept. of Clinical Research
Weston, Florida, 33331, United States
Indiana University Cancer Pavilion, Div. of Hematology-Oncology, Hematological Malignancy Program/Immunology
Indianapolis, Indiana, 46202, United States
Johns Hopkins University Medical Center
Baltimore, Maryland, 21205, United States
National Heart, Blood, and Lung Institute, National Institutes of Health
Bethesda, Maryland, 20892, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Washington University School of Medicine, Dept. of Internal Medicine/Division of Hematology
St Louis, Missouri, 63110-1093, United States
NYU Clinical Cancer Center
New York, New York, 10016, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10021, United States
Duke University Health System, Division of Cell Therapy, Heme Malignancies Program
Durham, North Carolina, 27710, United States
Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
Philadelphia, Pennsylvania, 19104, United States
Princess Alexandra Hospital, Oncology Haematology Radiation Department
Woolloongabba, Queensland, 4102, Australia
Queen Elizabeth Hospital, Dept. of Haematology
Woodville South, South Australia, 5011, Australia
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
Parkville, Victoria, 3050, Australia
The Royal Perth Hosptial, Department of Haematology/Level 2
Perth, Western Australia, 6000, Australia
Ucl St. Luc, Hematology Department
Brussels, 1200, Belgium
University of Alberta, Cross Cancer Institute
Edmonton, Alberta, T6G 2B7, Canada
Hospital De L'Hotel Dieu, Hematologie et Oncologie Medicale
Cedex, Paris, 04 75181, France
Hopital Saint-Louis, Centre G. Hayem-Secteur Bleu Porte 7, Laboratoire De Pathologie/Greffe de Moelle
Cedex, Paris, 10 75475, France
Universitatsklinikum Essen, Zentrum fur Innere Medizin
Essen, D-45147, Germany
Universitatsklinik Greifswald, Innere Medizin C - Hamato-Onkologie
Greifswald, D-17487, Germany
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
Hanover, D-30625, Germany
Saarland University Medical School, Internal Medicine 1
Homburg/Saar, D-66421, Germany
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionsmedizin des Universitatskinikums Ulm
Ulm, D-89081, Germany
St. James Hospital, Haematology Department
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
Universitar 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, Dept. of Hematology
Nijmegen, GA, 6525, Netherlands
Lund University Hospital, Department of Internal Medicine, Section of Hematology
Lund, SE-22185, Sweden
Umea University Hospital, Dept. of Internal Medicine 3, Section for Hematology
Umeå, 90185, Sweden
Universitatsklinik Basel, Division of Hematology
Basel, CH, 4031, Switzerland
Royal Cornwall Hospital, Haematology Dept.
Truro, Cornwall, TR1 3LJ, United Kingdom
Belfast City Hospital, Dept. of Haematology C Floor
Belfast, BT9 7AB, United Kingdom
Leeds General Infirmary, D Floor Brotherton Wing
Leeds, LS1 3EX, United Kingdom
St. George's Hospital, Department of Haematology
London, SW17 OQT, United Kingdom
Related Publications (2)
Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, Roth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. doi: 10.1056/NEJMoa061648.
PMID: 16990386RESULTHill A, Rother RP, Wang X, Morris SM Jr, Quinn-Senger K, Kelly R, Richards SJ, Bessler M, Bell L, Hillmen P, Gladwin MT. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2010 May;149(3):414-25. doi: 10.1111/j.1365-2141.2010.08096.x. Epub 2010 Mar 8.
PMID: 20230403DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Purpose
- ECT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
July 18, 2005
First Posted
July 22, 2005
Study Start
October 1, 2004
Study Completion
January 1, 2006
Last Updated
December 4, 2006
Record last verified: 2006-11