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

Hemoglobinuria, Paroxysmal Clinical Trial

Official title:

Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00122304
• Other study ID #: SHEPHERD
• Secondary ID: C04-002
• Status: Completed
• Phase: Phase 3
• First received: July 19, 2005
• Last updated: February 20, 2007
• Start date: December 2004
• Est. completion date: November 2006

Study information

• Verified date: February 2007
• 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 of eculizumab in patients with transfusion-dependent hemolytic PNH

Other known NCT identifiers

• NCT00133120

Recruitment information / eligibility

• Status: Completed
• Enrollment: 85
• Est. completion date: November 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

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
Australia	The Royal Perth Hospital, Dept. of Haematology/Level 2	Perth	Western Australia
Australia	Royal North Shore Hospital, Haematology Department	St. Leonard	New South Wales
Australia	The Queen Elizabeth Hospital, Haematology/Oncology Department	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
Canada	London Regional Cancer Centre, Clinical Research Unit Room C3080	London	Ontario
France	Hopital Saint-Louis, Centre d'investigation Clinique	Paris	Cedex
Germany	Universitatsklinikum Essen, Zentrum fur Innere Medizin	Essen
Germany	Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin	Hannover
Germany	Universitatskliniken des Saarlandes, Innere Medizin 1	Homburg/Saar
Germany	Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm	Ulm
Ireland	St. James Hospital, Haematology Dept., Cancer Clinical Trial Office	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	Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico	Napoli
Italy	Ospedale San Bortolo, Divisione di Ematologia	Vicenza
Netherlands	UMC St. Radboud, Department of Hematology	Nijmegen
Spain	Hospital Clinic i Provincial, Servicio de Hematologia	Barcelona
Spain	Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia	Barcelona
Spain	Hospital De La Paz, Servicio de Hematologia	Madrid
Sweden	Stockholm South Hospital, Division of Hematology	Stockholm
Sweden	University Hospital, Dept. of Haematology	Uppsala
Switzerland	Kantonsspital Basel, Abteilung fuer Haematologie	Basel
United Kingdom	Leeds General Infirmary, D Floor Brotherton Wing	Leeds
United Kingdom	St. Georges Hospital, Department of Haematology	London
United Kingdom	Royal Cornwall Hospital	Truro	Cornwall
United States	Johns Hopkins University Medical Center	Baltimore	Maryland
United States	National Heart, Lung, and Blood Institute, National Institutes of Health	Bethesda	Maryland
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Roswell Park Cancer Institute	Buffalo	New York
United States	The Cleveland Clinic Foundation	Cleveland	Ohio
United States	Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program	Durham	North Carolina
United States	Hartford Hospital, Cancer Clinical Research Office	Hartford	Connecticut
United States	Indiana University Cancer Pavilion	Indianapolis	Indiana
United States	Norris Comprehensive Cancer Center	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	OHSU Center for Hematologic Malignancies	Portland	Oregon
United States	Mayo Clinic, Division of Hematology	Rochester	Minnesota
United States	University of Utah, Hematology-BMT Department	Salt Lake City	Utah
United States	Washington University Medical Center, Department of Internal Medicine/Division of Hematology	St. Louis	Missouri
United States	Stanford University Medical Center, Division of Hematology	Stanford	California
United States	Cooper University Hospital, Cooper Cancer Institute	Voorhees	New Jersey
United States	Cleveland Clinic Florida, 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,  Spain,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Primary	Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
Secondary	Hemolysis measured by the change of LDH from baseline;
Secondary	Quality of Life