Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Hemoglobinuria, Nocturnal Clinical Trial

Official title:

Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00122317
• Other study ID #: E05-001
• Status: Completed
• Phase: Phase 3
• First received: July 20, 2005
• Last updated: February 11, 2018
• Start date: May 2005
• Est. completion date: October 2008

Study information

• Verified date: February 2018
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.

Description:

An open-label extension study to evaluate long-term safety of eculizumab in PNH patients who had completed the TRIUMPH (C04-001), SHEPHERD (C04-002), and X03-001 studies.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 187
• Est. completion date: October 2008
• Est. primary completion date: September 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Patients who have fully completed the TRIUMPH, SHEPHERD, or X03-001 studies

• TRIUMPH patients who have discontinued receiving investigational drug prior to the last visit of the study due to lack of efficacy or exacerbation of symptoms of PNH and have completed all monthly safety and efficacy procedures

• Patient must be willing and able to give written informed consent

• Patient must avoid conception during the trial

Exclusion Criteria:

• Patients who have terminated early from the SHEPHERD or X03-001 studies

• Patients who have terminated early from the TRIUMPH study due to an adverse event

• Female who is pregnant, breast feeding, or intending to conceive during the course of the study

• Any condition that could increase the patient's risk by participating in the study or could confound the outcome of the study

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Hemoglobinuria, Nocturnal

Intervention

Drug:
• eculizumab

Locations

Country	Name	City	State
Australia	Royal Melbourne Hospital, Department of Clinical Haematology & Medical Oncology	Parkville
Australia	The Royal Perth Hospital, Department of Haematology/Level 2	Perth	Western Australia
Australia	Royal North Shore Hospital, Haematology Department	Saint Leonards	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 Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm	Ulm
Ireland	St. James's Hospital, Cancer Clinical Trial Office	Dublin
Italy	Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica	Firenze
Italy	Ospedale San Martino, Department 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	GA
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
United Kingdom	Leeds General Infirmary, D Floor Brotherton Wing	Leeds
United Kingdom	St. George's Hospital, Department of Haematology	London
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	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	Indianapolis University Cancer Center	Indianapolis	Indiana
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 & Thombosis Program	Philadelphia	Pennsylvania
United States	Mayo Clinic, Divison of Hematology	Rochester	Minnesota
United States	Washington University, Department of Internal Medicine/Division of Hematology	Saint Louis	Missouri
United States	Stanford University Medical Center, Division of Hematology	Stanford	California
United States	Cleveland Clinic Florida, Department 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,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment-emergent Adverse Events		From time of consent to a maximum of 2.5 years of study treatment
Secondary	Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve		From time of first infusion through 24 months of study treatment
Secondary	Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline	The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.	From time of first infusion through 24 months of study treatment
Secondary	Incidence of Thrombosis After Eculizumab Infusion	Thrombosis was defined as occurrence of major adverse vascular events	From time of first ever dose through last dose (up to 24 months of study treatment)