Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH
1 other identifier
interventional
187
12 countries
40
Brief Summary
The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started May 2005
Typical duration for phase_3
40 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2005
CompletedFirst Submitted
Initial submission to the registry
July 20, 2005
CompletedFirst Posted
Study publicly available on registry
July 22, 2005
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2008
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2008
CompletedResults Posted
Study results publicly available
June 5, 2017
CompletedMarch 13, 2018
February 1, 2018
3.3 years
July 20, 2005
April 24, 2017
February 11, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Incidence of Treatment-emergent Adverse Events
From time of consent to a maximum of 2.5 years of study treatment
Secondary Outcomes (3)
Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve
From time of first infusion through 24 months of study treatment
Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline
From time of first infusion through 24 months of study treatment
Incidence of Thrombosis After Eculizumab Infusion
From time of first ever dose through last dose (up to 24 months of study treatment)
Study Arms (1)
Eculizumab
EXPERIMENTAL600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (40)
Stanford University Medical Center, Division of Hematology
Stanford, California, 94305, United States
Hartford Hospital, Cancer Clinical Research Office
Hartford, Connecticut, 06102, United States
Cleveland Clinic Florida, Department of Clinical Research
Weston, Florida, 33331, United States
Indianapolis University Cancer Center
Indianapolis, Indiana, 46202, United States
Johns Hopkins University Medical Center
Baltimore, Maryland, 21205, United States
National Heart, Lung, and Blood Institute, National Institutes of Health
Bethesda, Maryland, 20892, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Mayo Clinic, Divison of Hematology
Rochester, Minnesota, 55902, United States
Washington University, Department of Internal Medicine/Division of Hematology
St Louis, Missouri, 63110, United States
NYU Clinical Cancer Center
New York, New York, 10016, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10017, United States
Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
Durham, North Carolina, 27710, United States
Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thombosis Program
Philadelphia, Pennsylvania, 19104, United States
Royal North Shore Hospital, Haematology Department
Saint Leonards, New South Wales, 2065, Australia
Princess Alexandra Hospital, Oncology Haematology Radiation Department
Woolloongabba, Queensland, 4102, Australia
The Queen Elizabeth Hospital, Haematology/Oncology Department
Woodville South, South Australia, 5011, Australia
The Royal Perth Hospital, Department of Haematology/Level 2
Perth, Western Australia, 6000, Australia
Royal Melbourne Hospital, Department of Clinical Haematology & Medical Oncology
Parkville, 3050, Australia
Ucl St. Luc, Hematology Department
Brussels, 1200, Belgium
University of Alberta, Cross Cancer Institute
Edmonton, Alberta, T6G 2B7, Canada
London Regional Cancer Centre, Clinical Research Unit Room C3080
London, Ontario, N6A4L6, Canada
Hopital Saint Louis, Centre d'investigation Clinique
Paris, Cedex, 10 75475, France
Universitatsklinikum Essen, Zentrum fur Innere Medizin
Essen, D-45147, Germany
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
Hanover, D-30625, Germany
Universitatskliniken des Saarlandes, Innere Medizin 1
Homburg/Saar, D-66421, Germany
Institut fur Klinische Transfusionmedizin und Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm
Ulm, D-89081, Germany
St. James's Hospital, Cancer Clinical Trial Office
Dublin, 8, Ireland
Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
Florence, 50139, Italy
Ospedale San Martino, Department of Hematology
Genova, I-16132, Italy
Ospedale Maggiore di Milano, Divisione di Ematologia
Milan, 35-20122, Italy
Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
Napoli, 5-80131, Italy
Ospedale San Bortolo, Divisione di Ematologia
Vicenza, 37-36100, Italy
UMC St. Radboud, Department of Hematology
Nijmegen, GA, 6525, Netherlands
Hospital Clinic i Provincial, Servicio de Hematologia
Barcelona, 08036, Spain
Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
Barcelona, 08916, Spain
Hospital De La Paz, Servicio de Hematologia
Madrid, 28046, Spain
Stockholm South Hospital, Division of Hematology
Stockholm, 118-83, Sweden
Leeds General Infirmary, D Floor Brotherton Wing
Leeds, LS1 3EX, United Kingdom
St. George's Hospital, Department of Haematology
London, SW17OQT, United Kingdom
Related Publications (1)
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.
PMID: 17702897DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Alexion Pharmaceuticals, Inc.
- Organization
- Alexion Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 20, 2005
First Posted
July 22, 2005
Study Start
May 1, 2005
Primary Completion
September 1, 2008
Study Completion
October 1, 2008
Last Updated
March 13, 2018
Results First Posted
June 5, 2017
Record last verified: 2018-02
Data Sharing
- IPD Sharing
- Will not share