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

February 20, 2007 updated by: Alexion Pharmaceuticals

Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment

85

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Parkville, Australia, 3050
        • Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
    • New South Wales
      • St. Leonard, New South Wales, Australia, 2065
        • Royal North Shore Hospital, Haematology Department
    • Queensland
      • Woolloongabba, Queensland, Australia, 4102
        • Princess Alexandra Hospital, Oncology Haematology Radiation Department
    • South Australia
      • Woodville South, South Australia, Australia, 5011
        • The Queen Elizabeth Hospital, Haematology/Oncology Department
    • Western Australia
      • Perth, Western Australia, Australia, 6000
        • The Royal Perth Hospital, Dept. of Haematology/Level 2
  • Belgium
      • Brussels, Belgium, 1200
        • Ucl St. Luc, Hematology Department
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2B7
        • University of Alberta, Cross Cancer Institute
    • Ontario
      • London, Ontario, Canada, N6A 4L6
        • London Regional Cancer Centre, Clinical Research Unit Room C3080
  • France
    • Cedex
      • Paris, Cedex, France, 10 75475
        • Hopital Saint-Louis, Centre d'investigation Clinique
  • Germany
      • Essen, Germany, D-45147
        • Universitatsklinikum Essen, Zentrum fur Innere Medizin
      • Hannover, Germany, D-30625
        • Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
      • Homburg/Saar, Germany, D-66421
        • Universitatskliniken des Saarlandes, Innere Medizin 1
      • Ulm, Germany, D-89081
        • Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm
  • Ireland
      • Dublin, Ireland, 8
        • St. James Hospital, Haematology Dept., Cancer Clinical Trial Office
  • Italy
      • Firenze, Italy, 50139
        • Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
      • Genova, Italy, I-16132
        • Ospedale San Martino, Dept. of Hematology
      • Milano, Italy, 35-20122
        • Ospedale Maggiore di Milano, Divisione di Ematologia
      • Napoli, Italy, 5-80131
        • Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
      • Vicenza, Italy, 37-36100
        • Ospedale San Bortolo, Divisione di Ematologia
  • Netherlands
      • Nijmegen, Netherlands, 6525 GA
        • UMC St. Radboud, Department of Hematology
  • Spain
      • Barcelona, Spain, 08036
        • Hospital Clinic I Provincial, Servicio de Hematologia
      • Barcelona, Spain, 08916
        • Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
      • Madrid, Spain, 28046
        • Hospital De La Paz, Servicio de Hematologia
  • Sweden
      • Stockholm, Sweden, 118-83
        • Stockholm South Hospital, Division of Hematology
      • Uppsala, Sweden, SE-751
        • University Hospital, Dept. of Haematology
  • Switzerland
      • Basel, Switzerland, CH-4031
        • Kantonsspital Basel, Abteilung fuer Haematologie
  • United Kingdom
      • Leeds, United Kingdom, LS1 3EX
        • Leeds General Infirmary, D Floor Brotherton Wing
      • London, United Kingdom, 17 OQT
        • St. Georges Hospital, Department of Haematology
    • Cornwall
      • Truro, Cornwall, United Kingdom, TR1 3LJ
        • Royal Cornwall Hospital
  • United States
    • California
      • Los Angeles, California, United States, 90033
        • Norris Comprehensive Cancer Center
      • Stanford, California, United States, 94305
        • Stanford University Medical Center, Division of Hematology
    • Connecticut
      • Hartford, Connecticut, United States, 06102
        • Hartford Hospital, Cancer Clinical Research Office
    • Florida
      • Weston, Florida, United States, 33331
        • Cleveland Clinic Florida, Dept. of Clinical Research
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University Cancer Pavilion
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Johns Hopkins University Medical Center
      • Bethesda, Maryland, United States, 20892
        • National Heart, Lung, and Blood Institute, National Institutes of Health
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic, Division of Hematology
    • Missouri
      • St. Louis, Missouri, United States, 63110
        • Washington University Medical Center, Department of Internal Medicine/Division of Hematology
    • New Jersey
      • Voorhees, New Jersey, United States, 08043
        • Cooper University Hospital, Cooper Cancer Institute
    • New York
      • Buffalo, New York, United States, 14263
        • Roswell Park Cancer Institute
      • New York, New York, United States, 10016
        • Nyu Clinical Cancer Center
      • New York, New York, United States, 10017
        • Memorial Sloan Kettering Cancer Center
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • The Cleveland Clinic Foundation
    • Oregon
      • Portland, Oregon, United States, 97239
        • OHSU Center for Hematologic Malignancies
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah, Hematology-BMT Department

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
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

Outcome Measure
Quality of Life
Hemolysis measured by the change of LDH from baseline;

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2004

Study Completion

November 1, 2006

Study Registration Dates

First Submitted

July 19, 2005

First Submitted That Met QC Criteria

July 19, 2005

First Posted (Estimate)

July 22, 2005

Study Record Updates

Last Update Posted (Estimate)

February 21, 2007

Last Update Submitted That Met QC Criteria

February 20, 2007

Last Verified

February 1, 2007

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SHEPHERD
  • C04-002

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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