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Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab

Primary Purpose

Hemoglobinuria, Paroxysmal

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
eculizumab
Sponsored by
Alexion
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemoglobinuria, Paroxysmal

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Norris Comprehensive Cancer Center
  • Stanford University Medical Center, Division of Hematology
  • Hartford Hospital, Cancer Clinical Research Office
  • Cleveland Clinic Florida, Dept. of Clinical Research
  • Indiana University Cancer Pavilion
  • Johns Hopkins University Medical Center
  • National Heart, Lung, and Blood Institute, National Institutes of Health
  • Massachusetts General Hospital
  • Mayo Clinic, Division of Hematology
  • Washington University Medical Center, Department of Internal Medicine/Division of Hematology
  • Cooper University Hospital, Cooper Cancer Institute
  • Roswell Park Cancer Institute
  • NYU Clinical Cancer Center
  • Memorial Sloan Kettering Cancer Center
  • Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
  • The Cleveland Clinic Foundation
  • OHSU Center for Hematologic Malignancies
  • Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
  • University of Utah, Hematology-BMT Department
  • Royal North Shore Hospital, Haematology Department
  • Princess Alexandra Hospital, Oncology Haematology Radiation Department
  • The Queen Elizabeth Hospital, Haematology/Oncology Department
  • The Royal Perth Hospital, Dept. of Haematology/Level 2
  • Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
  • Ucl St. Luc, Hematology Department
  • University of Alberta, Cross Cancer Institute
  • London Regional Cancer Centre, Clinical Research Unit Room C3080
  • Hopital Saint-Louis, Centre d'investigation Clinique
  • Universitatsklinikum Essen, Zentrum fur Innere Medizin
  • Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
  • Universitatskliniken des Saarlandes, Innere Medizin 1
  • Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm
  • St. James Hospital, Haematology Dept., Cancer Clinical Trial Office
  • Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
  • Ospedale San Martino, Dept. of Hematology
  • Ospedale Maggiore di Milano, Divisione di Ematologia
  • Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
  • Ospedale San Bortolo, Divisione di Ematologia
  • UMC St. Radboud, Department of Hematology
  • Hospital Clinic i Provincial, Servicio de Hematologia
  • Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
  • Hospital De La Paz, Servicio de Hematologia
  • Stockholm South Hospital, Division of Hematology
  • University Hospital, Dept. of Haematology
  • Kantonsspital Basel, Abteilung fuer Haematologie
  • Royal Cornwall Hospital
  • Leeds General Infirmary, D Floor Brotherton Wing
  • St. Georges Hospital, Department of Haematology

Outcomes

Primary Outcome Measures

Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.

Secondary Outcome Measures

Hemolysis measured by the change of LDH from baseline;
Quality of Life

Full Information

First Posted
July 19, 2005
Last Updated
February 20, 2007
Sponsor
Alexion
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1. Study Identification

Unique Protocol Identification Number
NCT00122304
Brief Title
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

2. Study Status

Record Verification Date
February 2007
Overall Recruitment Status
Completed
Study Start Date
December 2004 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
November 2006 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Alexion

4. Oversight

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemoglobinuria, Paroxysmal

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
85 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
eculizumab
Primary Outcome Measure Information:
Title
Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Title
Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
Secondary Outcome Measure Information:
Title
Hemolysis measured by the change of LDH from baseline;
Title
Quality of Life

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Norris Comprehensive Cancer Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Stanford University Medical Center, Division of Hematology
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Hartford Hospital, Cancer Clinical Research Office
City
Hartford
State/Province
Connecticut
ZIP/Postal Code
06102
Country
United States
Facility Name
Cleveland Clinic Florida, Dept. of Clinical Research
City
Weston
State/Province
Florida
ZIP/Postal Code
33331
Country
United States
Facility Name
Indiana University Cancer Pavilion
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Johns Hopkins University Medical Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
National Heart, Lung, and Blood Institute, National Institutes of Health
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Mayo Clinic, Division of Hematology
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Washington University Medical Center, Department of Internal Medicine/Division of Hematology
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Cooper University Hospital, Cooper Cancer Institute
City
Voorhees
State/Province
New Jersey
ZIP/Postal Code
08043
Country
United States
Facility Name
Roswell Park Cancer Institute
City
Buffalo
State/Province
New York
ZIP/Postal Code
14263
Country
United States
Facility Name
NYU Clinical Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10017
Country
United States
Facility Name
Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
The Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
OHSU Center for Hematologic Malignancies
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Utah, Hematology-BMT Department
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Royal North Shore Hospital, Haematology Department
City
St. Leonard
State/Province
New South Wales
ZIP/Postal Code
2065
Country
Australia
Facility Name
Princess Alexandra Hospital, Oncology Haematology Radiation Department
City
Woolloongabba
State/Province
Queensland
ZIP/Postal Code
4102
Country
Australia
Facility Name
The Queen Elizabeth Hospital, Haematology/Oncology Department
City
Woodville South
State/Province
South Australia
ZIP/Postal Code
5011
Country
Australia
Facility Name
The Royal Perth Hospital, Dept. of Haematology/Level 2
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6000
Country
Australia
Facility Name
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
City
Parkville
ZIP/Postal Code
3050
Country
Australia
Facility Name
Ucl St. Luc, Hematology Department
City
Brussels
ZIP/Postal Code
1200
Country
Belgium
Facility Name
University of Alberta, Cross Cancer Institute
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2B7
Country
Canada
Facility Name
London Regional Cancer Centre, Clinical Research Unit Room C3080
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 4L6
Country
Canada
Facility Name
Hopital Saint-Louis, Centre d'investigation Clinique
City
Paris
State/Province
Cedex
ZIP/Postal Code
10 75475
Country
France
Facility Name
Universitatsklinikum Essen, Zentrum fur Innere Medizin
City
Essen
ZIP/Postal Code
D-45147
Country
Germany
Facility Name
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
City
Hannover
ZIP/Postal Code
D-30625
Country
Germany
Facility Name
Universitatskliniken des Saarlandes, Innere Medizin 1
City
Homburg/Saar
ZIP/Postal Code
D-66421
Country
Germany
Facility Name
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm
City
Ulm
ZIP/Postal Code
D-89081
Country
Germany
Facility Name
St. James Hospital, Haematology Dept., Cancer Clinical Trial Office
City
Dublin
ZIP/Postal Code
8
Country
Ireland
Facility Name
Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
City
Firenze
ZIP/Postal Code
50139
Country
Italy
Facility Name
Ospedale San Martino, Dept. of Hematology
City
Genova
ZIP/Postal Code
I-16132
Country
Italy
Facility Name
Ospedale Maggiore di Milano, Divisione di Ematologia
City
Milano
ZIP/Postal Code
35-20122
Country
Italy
Facility Name
Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
City
Napoli
ZIP/Postal Code
5-80131
Country
Italy
Facility Name
Ospedale San Bortolo, Divisione di Ematologia
City
Vicenza
ZIP/Postal Code
37-36100
Country
Italy
Facility Name
UMC St. Radboud, Department of Hematology
City
Nijmegen
ZIP/Postal Code
6525 GA
Country
Netherlands
Facility Name
Hospital Clinic i Provincial, Servicio de Hematologia
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
City
Barcelona
ZIP/Postal Code
08916
Country
Spain
Facility Name
Hospital De La Paz, Servicio de Hematologia
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Stockholm South Hospital, Division of Hematology
City
Stockholm
ZIP/Postal Code
118-83
Country
Sweden
Facility Name
University Hospital, Dept. of Haematology
City
Uppsala
ZIP/Postal Code
SE-751
Country
Sweden
Facility Name
Kantonsspital Basel, Abteilung fuer Haematologie
City
Basel
ZIP/Postal Code
CH-4031
Country
Switzerland
Facility Name
Royal Cornwall Hospital
City
Truro
State/Province
Cornwall
ZIP/Postal Code
TR1 3LJ
Country
United Kingdom
Facility Name
Leeds General Infirmary, D Floor Brotherton Wing
City
Leeds
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Facility Name
St. Georges Hospital, Department of Haematology
City
London
ZIP/Postal Code
17 OQT
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab

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