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

This study has been completed.
Sponsor:
Information provided by:
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00122304
First received: July 19, 2005
Last updated: February 20, 2007
Last verified: February 2007
  Purpose

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


Condition Intervention Phase
Hemoglobinuria, Paroxysmal
Drug: eculizumab
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • 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:
  • Hemolysis measured by the change of LDH from baseline;
  • Quality of Life

Estimated Enrollment: 85
Study Start Date: December 2004
Estimated Study Completion Date: November 2006
  Eligibility

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

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00122304

  Hide Study Locations
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
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10017
NYU Clinical Cancer Center
New York, New York, United States, 10016
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
Australia
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
Parkville, Australia, 3050
Belgium
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
France
Hopital Saint-Louis, Centre d'investigation Clinique
Paris, Cedex, France, 10 75475
Germany
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
Ireland
St. James Hospital, Haematology Dept., Cancer Clinical Trial Office
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
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
Netherlands
UMC St. Radboud, Department of Hematology
Nijmegen, Netherlands, 6525 GA
Spain
Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
Barcelona, Spain, 08916
Hospital Clinic i Provincial, Servicio de Hematologia
Barcelona, Spain, 08036
Hospital De La Paz, Servicio de Hematologia
Madrid, Spain, 28046
Sweden
Stockholm South Hospital, Division of Hematology
Stockholm, Sweden, 118-83
University Hospital, Dept. of Haematology
Uppsala, Sweden, SE-751
Switzerland
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
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00122304     History of Changes
Obsolete Identifiers: NCT00133120
Other Study ID Numbers: SHEPHERD, C04-002
Study First Received: July 19, 2005
Last Updated: February 20, 2007
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 September 22, 2014