Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT ID: NCT00122317

Last Updated: 2018-03-13

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

187 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-05-31

Study Completion Date

2008-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

An open-label extension study to evaluate long-term safety of eculizumab in PNH patients who had completed the TRIUMPH (C04-001), SHEPHERD (C04-002), and X03-001 studies.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Paroxysmal Hemoglobinuria, Nocturnal

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Eculizumab

600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks

Group Type EXPERIMENTAL

eculizumab

Intervention Type DRUG

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

eculizumab

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Soliris

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Patients who have fully completed the TRIUMPH, SHEPHERD, or X03-001 studies
* TRIUMPH patients who have discontinued receiving investigational drug prior to the last visit of the study due to lack of efficacy or exacerbation of symptoms of PNH and have completed all monthly safety and efficacy procedures
* Patient must be willing and able to give written informed consent
* Patient must avoid conception during the trial

Exclusion Criteria

* Patients who have terminated early from the SHEPHERD or X03-001 studies
* Patients who have terminated early from the TRIUMPH study due to an adverse event
* Female who is pregnant, breast feeding, or intending to conceive during the course of the study
* Any condition that could increase the patient's risk by participating in the study or could confound the outcome of the study
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

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, Department of Clinical Research

Weston, Florida, United States

Site Status

Indianapolis University Cancer Center

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, Divison of Hematology

Rochester, Minnesota, United States

Site Status

Washington University, Department of Internal Medicine/Division of Hematology

St Louis, Missouri, 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

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thombosis Program

Philadelphia, Pennsylvania, United States

Site Status

Royal North Shore Hospital, Haematology Department

Saint Leonards, 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, Department of Haematology/Level 2

Perth, Western Australia, Australia

Site Status

Royal Melbourne Hospital, Department 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 Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm

Ulm, , Germany

Site Status

St. James's Hospital, 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, Department 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, GA, 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

Leeds General Infirmary, D Floor Brotherton Wing

Leeds, , United Kingdom

Site Status

St. George's Hospital, Department of Haematology

London, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

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

References

Explore related publications, articles, or registry entries linked to this study.

Hillmen P, Muus P, Duhrsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socie G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. doi: 10.1182/blood-2007-06-095646. Epub 2007 Aug 16.

Reference Type DERIVED
PMID: 17702897 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

E05-001

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Dynepo Long-Term Safety Study
NCT00514813 TERMINATED PHASE4
Danicopan Early Access Program
NCT05982938 AVAILABLE