Clinical trial NCT00122304: Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
Official title: Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemoglobinuria, Paroxysmal
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2005-07-22
Enrollment: 85
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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

Primary outcomes:
measure: Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
measure: Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
Secondary outcomes:
measure: Hemolysis measured by the change of LDH from baseline;
measure: Quality of Life
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Norris Comprehensive Cancer Center
state: California
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, Dept. of Clinical Research
state: Florida
city: Indianapolis
country: United States
facility: Indiana University Cancer Pavilion
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, Division of Hematology
state: Minnesota
city: St. Louis
country: United States
facility: Washington University Medical Center, Department of Internal Medicine/Division of Hematology
state: Missouri
city: Voorhees
country: United States
facility: Cooper University Hospital, Cooper Cancer Institute
state: New Jersey
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
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: The Cleveland Clinic Foundation
state: Ohio
city: Portland
country: United States
facility: OHSU Center for Hematologic Malignancies
state: Oregon
city: Philadelphia
country: United States
facility: Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
state: Pennsylvania
city: Salt Lake City
country: United States
facility: University of Utah, Hematology-BMT Department
state: Utah
city: St. Leonard
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, Dept. of Haematology/Level 2
state: Western Australia
city: Parkville
country: Australia
facility: Royal Melbourne Hospital, Dept. 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 Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm
city: Dublin
country: Ireland
facility: St. James Hospital, Haematology Dept., 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, Dept. 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
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: Uppsala
country: Sweden
facility: University Hospital, Dept. of Haematology
city: Basel
country: Switzerland
facility: Kantonsspital Basel, Abteilung fuer Haematologie
city: Truro
country: United Kingdom
facility: Royal Cornwall Hospital
state: Cornwall
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary, D Floor Brotherton Wing
city: London
country: United Kingdom
facility: St. Georges Hospital, Department of Haematology
Study officials:
References: