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Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

This study has been completed.
Sponsor:
Information provided by:
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00122330
First received: July 18, 2005
Last updated: November 30, 2006
Last verified: November 2006
History of Changes
  Purpose

The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.


Condition Intervention Phase
Hemoglobinuria, Paroxysmal
 Drug: eculizumab
 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Educational/Counseling/Training
Official Title: Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients

Resource links provided by NLM:

Drug Information available for: Eculizumab
U.S. FDA Resources

Further study details as provided by Alexion Pharmaceuticals:

Estimated Enrollment: 75
Study Start Date: October 2004
Estimated Study Completion Date: January 2006
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00122330

  Hide Study Locations
Locations
United States, California
City of Hope National Medical Center, Div. of Hematology & Bone Marrow Transplant
Duarte, California, United States, 91010-3000
University of California at Los Angeles
Los Angeles, California, United States, 90024
Scripps Cancer Center
San Diego, California, United States, 92121
Stanford University Medical Center, Division of Hematology
Stanford, California, United States, 94305-5821
United States, Connecticut
Hartford Hospital, Cancer Clinical Research Office
Hartford, Connecticut, United States, 06102
United States, Florida
Cleveland Clinic, Dept. of Clinical Research
Weston, Florida, United States, 33331
United States, Indiana
Indiana University Cancer Pavilion, Div. of Hematology-Oncology, Hematological Malignancy Program/Immunology
Indianapolis, Indiana, United States, 46202
United States, Maryland
Johns Hopkins University Medical Center
Baltimore, Maryland, United States, 21205
National Heart, Blood, and Lung Institute, National Institutes of Health
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Missouri
Washington University School of Medicine, Dept. of Internal Medicine/Division of Hematology
St. Louis, Missouri, United States, 63110-1093
United States, New York
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10021
NYU Clinical Cancer Center
New York, New York, United States, 10016
United States, North Carolina
Duke University Health System, Division of Cell Therapy, Heme Malignancies Program
Durham, North Carolina, United States, 27710
United States, Ohio
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
Philadelphia, Pennsylvania, United States, 19104
Australia, Queensland
Princess Alexandra Hospital, Oncology Haematology Radiation Department
Woolloongabba, Queensland, Australia, 4102
Australia, South Australia
Queen Elizabeth Hospital, Dept. of Haematology
Woodville South, South Australia, Australia, 5011
Australia, Victoria
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
Parkville, Victoria, Australia, 3050
Australia, Western Australia
The Royal Perth Hosptial, Department of Haematology/Level 2
Perth, Western Australia, Australia, 6000
Belgium
Ucl St. Luc, Hematology Department
Brussels, Belgium, 1200
Canada, Alberta
University of Alberta, Cross Cancer Institute
Edmonton, Alberta, Canada, T6G 2B7
France
Hospital De L'Hotel Dieu, Hematologie et Oncologie Medicale
Cedex, Paris, France, 04 75181
Hopital Saint-Louis, Centre G. Hayem-Secteur Bleu Porte 7, Laboratoire De Pathologie/Greffe de Moelle
Cedex, Paris, France, 10 75475
Germany
Universitatsklinikum Essen, Zentrum fur Innere Medizin
Essen, Germany, D-45147
Universitatsklinik Greifswald, Innere Medizin C - Hamato-Onkologie
Greifswald, Germany, D-17487
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
Hannover, Germany, D-30625
Saarland University Medical School, Internal Medicine 1
Homburg/Saar, Germany, D-66421
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionsmedizin des Universitatskinikums Ulm
Ulm, Germany, D-89081
Ireland
St. James Hospital, Haematology Department
Dublin, Ireland, 8
Italy
Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
Firenze, Italy, 50139
Ospedale San Martino, Dept. of Hematology
Genova, Italy, I-16132
Ospedale Maggiore di Milano, Divisione di Ematologia
Milano, Italy, 35-20122
Universitar degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
Napoli, Italy, 5-80131
Ospedale San Bortolo, Divisione di Ematologia
Vicenza, Italy, 37-36100
Netherlands
UMC St. Radboud, Dept. of Hematology
Nijmegen, GA, Netherlands, 6525
Sweden
Lund University Hospital, Department of Internal Medicine, Section of Hematology
Lund, Sweden, SE-22185
Umea University Hospital, Dept. of Internal Medicine 3, Section for Hematology
Umea, Sweden, 90185
Switzerland
Universitatsklinik Basel, Division of Hematology
Basel, CH, Switzerland, 4031
United Kingdom
Royal Cornwall Hospital, Haematology Dept.
Truro, Cornwall, United Kingdom, TR1 3LJ
Belfast City Hospital, Dept. of Haematology C Floor
Belfast, United Kingdom, BT9 7AB
Leeds General Infirmary, D Floor Brotherton Wing
Leeds, United Kingdom, LS1 3EX
St. George's Hospital, Department of Haematology
London, United Kingdom, SW17 OQT
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Publications:

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

ClinicalTrials.gov Identifier: NCT00122330     History of Changes
Other Study ID Numbers: TRIUMPH
Study First Received: July 18, 2005
Last Updated: November 30, 2006
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
 Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases

ClinicalTrials.gov processed this record on May 23, 2013