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

Hemoglobinuria, Paroxysmal Clinical Trial

Official title:

Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00122330
• Other study ID #: TRIUMPH
• Status: Completed
• Phase: Phase 3
• First received: July 18, 2005
• Last updated: November 30, 2006
• Start date: October 2004
• Est. completion date: January 2006

Study information

• Verified date: November 2006
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 75
• Est. completion date: January 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Educational/Counseling/Training

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal

Intervention

Drug:
• eculizumab

Locations

Country	Name	City	State
Australia	Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology	Parkville	Victoria
Australia	The Royal Perth Hosptial, Department of Haematology/Level 2	Perth	Western Australia
Australia	Queen Elizabeth Hospital, Dept. of Haematology	Woodville South	South Australia
Australia	Princess Alexandra Hospital, Oncology Haematology Radiation Department	Woolloongabba	Queensland
Belgium	Ucl St. Luc, Hematology Department	Brussels
Canada	University of Alberta, Cross Cancer Institute	Edmonton	Alberta
France	Hopital Saint-Louis, Centre G. Hayem-Secteur Bleu Porte 7, Laboratoire De Pathologie/Greffe de Moelle	Cedex	Paris
France	Hospital De L'Hotel Dieu, Hematologie et Oncologie Medicale	Cedex	Paris
Germany	Universitatsklinikum Essen, Zentrum fur Innere Medizin	Essen
Germany	Universitatsklinik Greifswald, Innere Medizin C - Hamato-Onkologie	Greifswald
Germany	Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin	Hannover
Germany	Saarland University Medical School, Internal Medicine 1	Homburg/Saar
Germany	Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionsmedizin des Universitatskinikums Ulm	Ulm
Ireland	St. James Hospital, Haematology Department	Dublin
Italy	Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica	Firenze
Italy	Ospedale San Martino, Dept. of Hematology	Genova
Italy	Ospedale Maggiore di Milano, Divisione di Ematologia	Milano
Italy	Universitar degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico	Napoli
Italy	Ospedale San Bortolo, Divisione di Ematologia	Vicenza
Netherlands	UMC St. Radboud, Dept. of Hematology	Nijmegen	GA
Sweden	Lund University Hospital, Department of Internal Medicine, Section of Hematology	Lund
Sweden	Umea University Hospital, Dept. of Internal Medicine 3, Section for Hematology	Umea
Switzerland	Universitatsklinik Basel, Division of Hematology	Basel	CH
United Kingdom	Belfast City Hospital, Dept. of Haematology C Floor	Belfast
United Kingdom	Leeds General Infirmary, D Floor Brotherton Wing	Leeds
United Kingdom	St. George's Hospital, Department of Haematology	London
United Kingdom	Royal Cornwall Hospital, Haematology Dept.	Truro	Cornwall
United States	Johns Hopkins University Medical Center	Baltimore	Maryland
United States	National Heart, Blood, and Lung Institute, National Institutes of Health	Bethesda	Maryland
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	City of Hope National Medical Center, Div. of Hematology & Bone Marrow Transplant	Duarte	California
United States	Duke University Health System, Division of Cell Therapy, Heme Malignancies Program	Durham	North Carolina
United States	Hartford Hospital, Cancer Clinical Research Office	Hartford	Connecticut
United States	Indiana University Cancer Pavilion, Div. of Hematology-Oncology, Hematological Malignancy Program/Immunology	Indianapolis	Indiana
United States	University of California at Los Angeles	Los Angeles	California
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	NYU Clinical Cancer Center	New York	New York
United States	Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program	Philadelphia	Pennsylvania
United States	Scripps Cancer Center	San Diego	California
United States	Washington University School of Medicine, Dept. of Internal Medicine/Division of Hematology	St. Louis	Missouri
United States	Stanford University Medical Center, Division of Hematology	Stanford	California
United States	Cleveland Clinic, Dept. of Clinical Research	Weston	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada,  France,  Germany,  Ireland,  Italy,  Netherlands,  Sweden,  Switzerland,  United Kingdom, 

References & Publications (1)

Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, Röth 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 i — View Citation