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

NCT ID: NCT00122304

Last Updated: 2007-02-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

85 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-12-31

Study Completion Date

2006-11-30

Brief Summary

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

Detailed Description

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Conditions

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Hemoglobinuria, Paroxysmal

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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eculizumab

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Locations

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Norris Comprehensive Cancer Center

Los Angeles, California, United States

Site Status

Stanford University Medical Center, Division of Hematology

Stanford, California, United States

Site Status

Hartford Hospital, Cancer Clinical Research Office

Hartford, Connecticut, United States

Site Status

Cleveland Clinic Florida, Dept. of Clinical Research

Weston, Florida, United States

Site Status

Indiana University Cancer Pavilion

Indianapolis, Indiana, United States

Site Status

Johns Hopkins University Medical Center

Baltimore, Maryland, United States

Site Status

National Heart, Lung, and Blood Institute, National Institutes of Health

Bethesda, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Mayo Clinic, Division of Hematology

Rochester, Minnesota, United States

Site Status

Washington University Medical Center, Department of Internal Medicine/Division of Hematology

St Louis, Missouri, United States

Site Status

Cooper University Hospital, Cooper Cancer Institute

Voorhees Township, New Jersey, United States

Site Status

Roswell Park Cancer Institute

Buffalo, New York, United States

Site Status

NYU Clinical Cancer Center

New York, New York, United States

Site Status

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status

Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program

Durham, North Carolina, United States

Site Status

The Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

OHSU Center for Hematologic Malignancies

Portland, Oregon, United States

Site Status

Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program

Philadelphia, Pennsylvania, United States

Site Status

University of Utah, Hematology-BMT Department

Salt Lake City, Utah, United States

Site Status

Royal North Shore Hospital, Haematology Department

Saint Leonard, New South Wales, Australia

Site Status

Princess Alexandra Hospital, Oncology Haematology Radiation Department

Woolloongabba, Queensland, Australia

Site Status

The Queen Elizabeth Hospital, Haematology/Oncology Department

Woodville South, South Australia, Australia

Site Status

The Royal Perth Hospital, Dept. of Haematology/Level 2

Perth, Western Australia, Australia

Site Status

Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology

Parkville, , Australia

Site Status

Ucl St. Luc, Hematology Department

Brussels, , Belgium

Site Status

University of Alberta, Cross Cancer Institute

Edmonton, Alberta, Canada

Site Status

London Regional Cancer Centre, Clinical Research Unit Room C3080

London, Ontario, Canada

Site Status

Hopital Saint-Louis, Centre d'investigation Clinique

Paris, Cedex, France

Site Status

Universitatsklinikum Essen, Zentrum fur Innere Medizin

Essen, , Germany

Site Status

Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin

Hanover, , Germany

Site Status

Universitatskliniken des Saarlandes, Innere Medizin 1

Homburg/Saar, , Germany

Site Status

Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm

Ulm, , Germany

Site Status

St. James Hospital, Haematology Dept., Cancer Clinical Trial Office

Dublin, , Ireland

Site Status

Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica

Florence, , Italy

Site Status

Ospedale San Martino, Dept. of Hematology

Genova, , Italy

Site Status

Ospedale Maggiore di Milano, Divisione di Ematologia

Milan, , Italy

Site Status

Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico

Napoli, , Italy

Site Status

Ospedale San Bortolo, Divisione di Ematologia

Vicenza, , Italy

Site Status

UMC St. Radboud, Department of Hematology

Nijmegen, , Netherlands

Site Status

Hospital Clinic i Provincial, Servicio de Hematologia

Barcelona, , Spain

Site Status

Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia

Barcelona, , Spain

Site Status

Hospital De La Paz, Servicio de Hematologia

Madrid, , Spain

Site Status

Stockholm South Hospital, Division of Hematology

Stockholm, , Sweden

Site Status

University Hospital, Dept. of Haematology

Uppsala, , Sweden

Site Status

Kantonsspital Basel, Abteilung fuer Haematologie

Basel, , Switzerland

Site Status

Royal Cornwall Hospital

Truro, Cornwall, United Kingdom

Site Status

Leeds General Infirmary, D Floor Brotherton Wing

Leeds, , United Kingdom

Site Status

St. Georges Hospital, Department of Haematology

London, , United Kingdom

Site Status

Countries

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United States Australia Belgium Canada France Germany Ireland Italy Netherlands Spain Sweden Switzerland United Kingdom

Other Identifiers

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C04-002

Identifier Type: -

Identifier Source: secondary_id

SHEPHERD

Identifier Type: -

Identifier Source: org_study_id

NCT00133120

Identifier Type: -

Identifier Source: nct_alias

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