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Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

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 educational/counseling/training trial for Hemoglobinuria, Paroxysmal

Eligibility Criteria

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

Inclusion Criteria: Must have required at least 4 transfusions in the past 12 months PNH type III red blood cell (RBC) clone by flow cytometry of >10% Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal Platelet count > 100,000/mm3 Patient taking erythropoietin must be on a stable dose for at least 26 weeks Patient taking immunosuppressants must be on a stable dose for at least 26 weeks Patient taking corticosteroids must be on a stable dose for at least 4 weeks Patient taking coumadin must be at a stable INR for at least 4 weeks Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks Willing and able to give written informed consent Must avoid conception Exclusion Criteria: Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl Absolute neutrophil count <500/ul Active bacterial infection Hereditary complement deficiency Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days Pregnant, breast-feeding, or intending to conceive History of meningococcal disease History of bone marrow transplantation

Sites / Locations

  • City of Hope National Medical Center, Div. of Hematology & Bone Marrow Transplant
  • University of California at Los Angeles
  • Scripps Cancer Center
  • Stanford University Medical Center, Division of Hematology
  • Hartford Hospital, Cancer Clinical Research Office
  • Cleveland Clinic, Dept. of Clinical Research
  • Indiana University Cancer Pavilion, Div. of Hematology-Oncology, Hematological Malignancy Program/Immunology
  • Johns Hopkins University Medical Center
  • National Heart, Blood, and Lung Institute, National Institutes of Health
  • Massachusetts General Hospital
  • Washington University School of Medicine, Dept. of Internal Medicine/Division of Hematology
  • NYU Clinical Cancer Center
  • Memorial Sloan Kettering Cancer Center
  • Duke University Health System, Division of Cell Therapy, Heme Malignancies Program
  • Cleveland Clinic Foundation
  • Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
  • Princess Alexandra Hospital, Oncology Haematology Radiation Department
  • Queen Elizabeth Hospital, Dept. of Haematology
  • Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
  • The Royal Perth Hosptial, Department of Haematology/Level 2
  • Ucl St. Luc, Hematology Department
  • University of Alberta, Cross Cancer Institute
  • Hospital De L'Hotel Dieu, Hematologie et Oncologie Medicale
  • Hopital Saint-Louis, Centre G. Hayem-Secteur Bleu Porte 7, Laboratoire De Pathologie/Greffe de Moelle
  • Universitatsklinikum Essen, Zentrum fur Innere Medizin
  • Universitatsklinik Greifswald, Innere Medizin C - Hamato-Onkologie
  • Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
  • Saarland University Medical School, Internal Medicine 1
  • Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionsmedizin des Universitatskinikums Ulm
  • St. James Hospital, Haematology Department
  • Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
  • Ospedale San Martino, Dept. of Hematology
  • Ospedale Maggiore di Milano, Divisione di Ematologia
  • Universitar degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
  • Ospedale San Bortolo, Divisione di Ematologia
  • UMC St. Radboud, Dept. of Hematology
  • Lund University Hospital, Department of Internal Medicine, Section of Hematology
  • Umea University Hospital, Dept. of Internal Medicine 3, Section for Hematology
  • Universitatsklinik Basel, Division of Hematology
  • Royal Cornwall Hospital, Haematology Dept.
  • Belfast City Hospital, Dept. of Haematology C Floor
  • Leeds General Infirmary, D Floor Brotherton Wing
  • St. George's Hospital, Department of Haematology

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
July 18, 2005
Last Updated
November 30, 2006
Sponsor
Alexion
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1. Study Identification

Unique Protocol Identification Number
NCT00122330
Brief Title
Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Official Title
Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
Study Type
Interventional

2. Study Status

Record Verification Date
November 2006
Overall Recruitment Status
Completed
Study Start Date
October 2004 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
January 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 and efficacy of eculizumab in transfusion dependent patients with 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
Educational/Counseling/Training
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
Double
Allocation
Randomized
Enrollment
75 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
eculizumab

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Must have required at least 4 transfusions in the past 12 months PNH type III red blood cell (RBC) clone by flow cytometry of >10% Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal Platelet count > 100,000/mm3 Patient taking erythropoietin must be on a stable dose for at least 26 weeks Patient taking immunosuppressants must be on a stable dose for at least 26 weeks Patient taking corticosteroids must be on a stable dose for at least 4 weeks Patient taking coumadin must be at a stable INR for at least 4 weeks Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks Willing and able to give written informed consent Must avoid conception Exclusion Criteria: Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl Absolute neutrophil count <500/ul Active bacterial infection Hereditary complement deficiency Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days Pregnant, breast-feeding, or intending to conceive History of meningococcal disease History of bone marrow transplantation
Facility Information:
Facility Name
City of Hope National Medical Center, Div. of Hematology & Bone Marrow Transplant
City
Duarte
State/Province
California
ZIP/Postal Code
91010-3000
Country
United States
Facility Name
University of California at Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90024
Country
United States
Facility Name
Scripps Cancer Center
City
San Diego
State/Province
California
ZIP/Postal Code
92121
Country
United States
Facility Name
Stanford University Medical Center, Division of Hematology
City
Stanford
State/Province
California
ZIP/Postal Code
94305-5821
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, Dept. of Clinical Research
City
Weston
State/Province
Florida
ZIP/Postal Code
33331
Country
United States
Facility Name
Indiana University Cancer Pavilion, Div. of Hematology-Oncology, Hematological Malignancy Program/Immunology
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, Blood, and Lung 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
Washington University School of Medicine, Dept. of Internal Medicine/Division of Hematology
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63110-1093
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
10021
Country
United States
Facility Name
Duke University Health System, Division of Cell Therapy, Heme Malignancies Program
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
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
Princess Alexandra Hospital, Oncology Haematology Radiation Department
City
Woolloongabba
State/Province
Queensland
ZIP/Postal Code
4102
Country
Australia
Facility Name
Queen Elizabeth Hospital, Dept. of Haematology
City
Woodville South
State/Province
South Australia
ZIP/Postal Code
5011
Country
Australia
Facility Name
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
The Royal Perth Hosptial, Department of Haematology/Level 2
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6000
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
Hospital De L'Hotel Dieu, Hematologie et Oncologie Medicale
City
Cedex
State/Province
Paris
ZIP/Postal Code
04 75181
Country
France
Facility Name
Hopital Saint-Louis, Centre G. Hayem-Secteur Bleu Porte 7, Laboratoire De Pathologie/Greffe de Moelle
City
Cedex
State/Province
Paris
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
Universitatsklinik Greifswald, Innere Medizin C - Hamato-Onkologie
City
Greifswald
ZIP/Postal Code
D-17487
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
Saarland University Medical School, Internal Medicine 1
City
Homburg/Saar
ZIP/Postal Code
D-66421
Country
Germany
Facility Name
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionsmedizin des Universitatskinikums Ulm
City
Ulm
ZIP/Postal Code
D-89081
Country
Germany
Facility Name
St. James Hospital, Haematology Department
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
Universitar 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, Dept. of Hematology
City
Nijmegen
State/Province
GA
ZIP/Postal Code
6525
Country
Netherlands
Facility Name
Lund University Hospital, Department of Internal Medicine, Section of Hematology
City
Lund
ZIP/Postal Code
SE-22185
Country
Sweden
Facility Name
Umea University Hospital, Dept. of Internal Medicine 3, Section for Hematology
City
Umea
ZIP/Postal Code
90185
Country
Sweden
Facility Name
Universitatsklinik Basel, Division of Hematology
City
Basel
State/Province
CH
ZIP/Postal Code
4031
Country
Switzerland
Facility Name
Royal Cornwall Hospital, Haematology Dept.
City
Truro
State/Province
Cornwall
ZIP/Postal Code
TR1 3LJ
Country
United Kingdom
Facility Name
Belfast City Hospital, Dept. of Haematology C Floor
City
Belfast
ZIP/Postal Code
BT9 7AB
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. George's Hospital, Department of Haematology
City
London
ZIP/Postal Code
SW17 OQT
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
16990386
Citation
Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, Roth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. doi: 10.1056/NEJMoa061648.
Results Reference
result
PubMed Identifier
20230403
Citation
Hill A, Rother RP, Wang X, Morris SM Jr, Quinn-Senger K, Kelly R, Richards SJ, Bessler M, Bell L, Hillmen P, Gladwin MT. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2010 May;149(3):414-25. doi: 10.1111/j.1365-2141.2010.08096.x. Epub 2010 Mar 8.
Results Reference
derived

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Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

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