Clinical trial NCT00122317: Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official title: Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Paroxysmal Hemoglobinuria, Nocturnal
Keywords: transfusion dependent
paroxysmal nocturnal hemoglobinuria
hemolytic
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2005-07-22
Enrollment: 187
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Incidence of Treatment-emergent Adverse Events
timeFrame: From time of consent to a maximum of 2.5 years of study treatment
Secondary outcomes:
measure: Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve
timeFrame: From time of first infusion through 24 months of study treatment
measure: Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline
timeFrame: From time of first infusion through 24 months of study treatment
measure: Incidence of Thrombosis After Eculizumab Infusion
timeFrame: From time of first ever dose through last dose (up to 24 months of study treatment)
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University Medical Center, Division of Hematology
state: California
city: Hartford
country: United States
facility: Hartford Hospital, Cancer Clinical Research Office
state: Connecticut
city: Weston
country: United States
facility: Cleveland Clinic Florida, Department of Clinical Research
state: Florida
city: Indianapolis
country: United States
facility: Indianapolis University Cancer Center
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins University Medical Center
state: Maryland
city: Bethesda
country: United States
facility: National Heart, Lung, and Blood Institute, National Institutes of Health
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic, Divison of Hematology
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University, Department of Internal Medicine/Division of Hematology
state: Missouri
city: New York
country: United States
facility: NYU Clinical Cancer Center
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Philadelphia
country: United States
facility: Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thombosis Program
state: Pennsylvania
city: Saint Leonards
country: Australia
facility: Royal North Shore Hospital, Haematology Department
state: New South Wales
city: Woolloongabba
country: Australia
facility: Princess Alexandra Hospital, Oncology Haematology Radiation Department
state: Queensland
city: Woodville South
country: Australia
facility: The Queen Elizabeth Hospital, Haematology/Oncology Department
state: South Australia
city: Perth
country: Australia
facility: The Royal Perth Hospital, Department of Haematology/Level 2
state: Western Australia
city: Parkville
country: Australia
facility: Royal Melbourne Hospital, Department of Clinical Haematology & Medical Oncology
city: Brussels
country: Belgium
facility: Ucl St. Luc, Hematology Department
city: Edmonton
country: Canada
facility: University of Alberta, Cross Cancer Institute
state: Alberta
city: London
country: Canada
facility: London Regional Cancer Centre, Clinical Research Unit Room C3080
state: Ontario
city: Paris
country: France
facility: Hopital Saint Louis, Centre d'investigation Clinique
state: Cedex
city: Essen
country: Germany
facility: Universitatsklinikum Essen, Zentrum fur Innere Medizin
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
city: Homburg/Saar
country: Germany
facility: Universitatskliniken des Saarlandes, Innere Medizin 1
city: Ulm
country: Germany
facility: Institut fur Klinische Transfusionmedizin und Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm
city: Dublin
country: Ireland
facility: St. James's Hospital, Cancer Clinical Trial Office
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
city: Genova
country: Italy
facility: Ospedale San Martino, Department of Hematology
city: Milano
country: Italy
facility: Ospedale Maggiore di Milano, Divisione di Ematologia
city: Napoli
country: Italy
facility: Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
city: Vicenza
country: Italy
facility: Ospedale San Bortolo, Divisione di Ematologia
city: Nijmegen
country: Netherlands
facility: UMC St. Radboud, Department of Hematology
state: GA
city: Barcelona
country: Spain
facility: Hospital Clinic i Provincial, Servicio de Hematologia
city: Barcelona
country: Spain
facility: Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
city: Madrid
country: Spain
facility: Hospital De La Paz, Servicio de Hematologia
city: Stockholm
country: Sweden
facility: Stockholm South Hospital, Division of Hematology
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary, D Floor Brotherton Wing
city: London
country: United Kingdom
facility: St. George's Hospital, Department of Haematology
Study officials:
References:
citation: 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.