Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00122304
Recruitment Status : Completed
First Posted : July 22, 2005
Last Update Posted : February 21, 2007
Information provided by:
Alexion Pharmaceuticals

Brief Summary:
The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH

Condition or disease Intervention/treatment Phase
Hemoglobinuria, Paroxysmal Drug: eculizumab Phase 3

Study Type : Interventional  (Clinical Trial)
Enrollment : 85 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
Study Start Date : December 2004
Study Completion Date : November 2006

Primary Outcome Measures :
  1. Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
  2. Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.

Secondary Outcome Measures :
  1. Hemolysis measured by the change of LDH from baseline;
  2. Quality of Life

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00122304

  Hide Study Locations
United States, California
Norris Comprehensive Cancer Center
Los Angeles, California, United States, 90033
Stanford University Medical Center, Division of Hematology
Stanford, California, United States, 94305
United States, Connecticut
Hartford Hospital, Cancer Clinical Research Office
Hartford, Connecticut, United States, 06102
United States, Florida
Cleveland Clinic Florida, Dept. of Clinical Research
Weston, Florida, United States, 33331
United States, Indiana
Indiana University Cancer Pavilion
Indianapolis, Indiana, United States, 46202
United States, Maryland
Johns Hopkins University Medical Center
Baltimore, Maryland, United States, 21205
National Heart, Lung, and Blood Institute, National Institutes of Health
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Minnesota
Mayo Clinic, Division of Hematology
Rochester, Minnesota, United States, 55905
United States, Missouri
Washington University Medical Center, Department of Internal Medicine/Division of Hematology
St. Louis, Missouri, United States, 63110
United States, New Jersey
Cooper University Hospital, Cooper Cancer Institute
Voorhees, New Jersey, United States, 08043
United States, New York
Roswell Park Cancer Institute
Buffalo, New York, United States, 14263
NYU Clinical Cancer Center
New York, New York, United States, 10016
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10017
United States, North Carolina
Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
Durham, North Carolina, United States, 27710
United States, Ohio
The Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
United States, Oregon
OHSU Center for Hematologic Malignancies
Portland, Oregon, United States, 97239
United States, Pennsylvania
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
Philadelphia, Pennsylvania, United States, 19104
United States, Utah
University of Utah, Hematology-BMT Department
Salt Lake City, Utah, United States, 84132
Australia, New South Wales
Royal North Shore Hospital, Haematology Department
St. Leonard, New South Wales, Australia, 2065
Australia, Queensland
Princess Alexandra Hospital, Oncology Haematology Radiation Department
Woolloongabba, Queensland, Australia, 4102
Australia, South Australia
The Queen Elizabeth Hospital, Haematology/Oncology Department
Woodville South, South Australia, Australia, 5011
Australia, Western Australia
The Royal Perth Hospital, Dept. of Haematology/Level 2
Perth, Western Australia, Australia, 6000
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
Parkville, Australia, 3050
Ucl St. Luc, Hematology Department
Brussels, Belgium, 1200
Canada, Alberta
University of Alberta, Cross Cancer Institute
Edmonton, Alberta, Canada, T6G 2B7
Canada, Ontario
London Regional Cancer Centre, Clinical Research Unit Room C3080
London, Ontario, Canada, N6A 4L6
Hopital Saint-Louis, Centre d'investigation Clinique
Paris, Cedex, France, 10 75475
Universitatsklinikum Essen, Zentrum fur Innere Medizin
Essen, Germany, D-45147
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
Hannover, Germany, D-30625
Universitatskliniken des Saarlandes, Innere Medizin 1
Homburg/Saar, Germany, D-66421
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm
Ulm, Germany, D-89081
St. James Hospital, Haematology Dept., Cancer Clinical Trial Office
Dublin, Ireland, 8
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
Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
Napoli, Italy, 5-80131
Ospedale San Bortolo, Divisione di Ematologia
Vicenza, Italy, 37-36100
UMC St. Radboud, Department of Hematology
Nijmegen, Netherlands, 6525 GA
Hospital Clinic i Provincial, Servicio de Hematologia
Barcelona, Spain, 08036
Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
Barcelona, Spain, 08916
Hospital De La Paz, Servicio de Hematologia
Madrid, Spain, 28046
Stockholm South Hospital, Division of Hematology
Stockholm, Sweden, 118-83
University Hospital, Dept. of Haematology
Uppsala, Sweden, SE-751
Kantonsspital Basel, Abteilung fuer Haematologie
Basel, Switzerland, CH-4031
United Kingdom
Royal Cornwall Hospital
Truro, Cornwall, United Kingdom, TR1 3LJ
Leeds General Infirmary, D Floor Brotherton Wing
Leeds, United Kingdom, LS1 3EX
St. Georges Hospital, Department of Haematology
London, United Kingdom, 17 OQT
Sponsors and Collaborators
Alexion Pharmaceuticals Identifier: NCT00122304     History of Changes
Obsolete Identifiers: NCT00133120
Other Study ID Numbers: SHEPHERD
First Posted: July 22, 2005    Key Record Dates
Last Update Posted: February 21, 2007
Last Verified: February 2007

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