Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Registration Number
- NCT00122317
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 187
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Eculizumab eculizumab 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
- Primary Outcome Measures
Name Time Method Incidence of Treatment-emergent Adverse Events From time of consent to a maximum of 2.5 years of study treatment
- Secondary Outcome Measures
Name Time Method Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve 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) Thrombosis was defined as occurrence of major adverse vascular events
Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline From time of first infusion through 24 months of study treatment The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.
Trial Locations
- Locations (40)
Hartford Hospital, Cancer Clinical Research Office
🇺🇸Hartford, Connecticut, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
The Royal Perth Hospital, Department of Haematology/Level 2
🇦🇺Perth, Western Australia, Australia
Ucl St. Luc, Hematology Department
🇧🇪Brussels, Belgium
NYU Clinical Cancer Center
🇺🇸New York, New York, United States
Royal Melbourne Hospital, Department of Clinical Haematology & Medical Oncology
🇦🇺Parkville, Australia
National Heart, Lung, and Blood Institute, National Institutes of Health
🇺🇸Bethesda, Maryland, United States
Universitatskliniken des Saarlandes, Innere Medizin 1
🇩🇪Homburg/Saar, Germany
Hospital Clinic i Provincial, Servicio de Hematologia
🇪🇸Barcelona, Spain
The Queen Elizabeth Hospital, Haematology/Oncology Department
🇦🇺Woodville South, South Australia, Australia
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thombosis Program
🇺🇸Philadelphia, Pennsylvania, United States
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
🇩🇪Hannover, Germany
Hopital Saint Louis, Centre d'investigation Clinique
🇫🇷Paris, Cedex, France
Princess Alexandra Hospital, Oncology Haematology Radiation Department
🇦🇺Woolloongabba, Queensland, Australia
Johns Hopkins University Medical Center
🇺🇸Baltimore, Maryland, United States
Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
🇺🇸Durham, North Carolina, United States
UMC St. Radboud, Department of Hematology
🇳🇱Nijmegen, GA, Netherlands
Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
🇮🇹Firenze, Italy
Stockholm South Hospital, Division of Hematology
🇸🇪Stockholm, Sweden
Ospedale San Martino, Department of Hematology
🇮🇹Genova, Italy
Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
🇪🇸Barcelona, Spain
Ospedale San Bortolo, Divisione di Ematologia
🇮🇹Vicenza, Italy
University of Alberta, Cross Cancer Institute
🇨🇦Edmonton, Alberta, Canada
London Regional Cancer Centre, Clinical Research Unit Room C3080
🇨🇦London, Ontario, Canada
Leeds General Infirmary, D Floor Brotherton Wing
🇬🇧Leeds, United Kingdom
Institut fur Klinische Transfusionmedizin und Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm
🇩🇪Ulm, Germany
Hospital De La Paz, Servicio de Hematologia
🇪🇸Madrid, Spain
Stanford University Medical Center, Division of Hematology
🇺🇸Stanford, California, United States
Mayo Clinic, Divison of Hematology
🇺🇸Rochester, Minnesota, United States
Indianapolis University Cancer Center
🇺🇸Indianapolis, Indiana, United States
Washington University, Department of Internal Medicine/Division of Hematology
🇺🇸Saint Louis, Missouri, United States
Cleveland Clinic Florida, Department of Clinical Research
🇺🇸Weston, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Memorial Sloan Kettering Cancer Center
🇺🇸New York, New York, United States
Universitatsklinikum Essen, Zentrum fur Innere Medizin
🇩🇪Essen, Germany
Ospedale Maggiore di Milano, Divisione di Ematologia
🇮🇹Milano, Italy
Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
🇮🇹Napoli, Italy
St. George's Hospital, Department of Haematology
🇬🇧London, United Kingdom
Royal North Shore Hospital, Haematology Department
🇦🇺Saint Leonards, New South Wales, Australia
St. James's Hospital, Cancer Clinical Trial Office
🇮🇪Dublin, Ireland