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

Primary Purpose

Paroxysmal Hemoglobinuria, Nocturnal

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

About this trial

This is an interventional treatment trial for Paroxysmal Hemoglobinuria, Nocturnal focused on measuring transfusion dependent, paroxysmal nocturnal hemoglobinuria, hemolytic

Eligibility Criteria

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

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

Sites / Locations

  • Stanford University Medical Center, Division of Hematology
  • Hartford Hospital, Cancer Clinical Research Office
  • Cleveland Clinic Florida, Department of Clinical Research
  • Indianapolis University Cancer Center
  • Johns Hopkins University Medical Center
  • National Heart, Lung, and Blood Institute, National Institutes of Health
  • Massachusetts General Hospital
  • Mayo Clinic, Divison of Hematology
  • Washington University, Department of Internal Medicine/Division of Hematology
  • NYU Clinical Cancer Center
  • Memorial Sloan Kettering Cancer Center
  • Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
  • Cleveland Clinic Foundation
  • Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thombosis Program
  • Royal North Shore Hospital, Haematology Department
  • Princess Alexandra Hospital, Oncology Haematology Radiation Department
  • The Queen Elizabeth Hospital, Haematology/Oncology Department
  • The Royal Perth Hospital, Department of Haematology/Level 2
  • Royal Melbourne Hospital, Department 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 Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm
  • St. James's Hospital, Cancer Clinical Trial Office
  • Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
  • Ospedale San Martino, Department 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
  • Leeds General Infirmary, D Floor Brotherton Wing
  • St. George's Hospital, Department of Haematology

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Eculizumab

Arm Description

600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks

Outcomes

Primary Outcome Measures

Incidence of Treatment-emergent Adverse Events

Secondary Outcome Measures

Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve
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.
Incidence of Thrombosis After Eculizumab Infusion
Thrombosis was defined as occurrence of major adverse vascular events

Full Information

First Posted
July 20, 2005
Last Updated
February 11, 2018
Sponsor
Alexion
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1. Study Identification

Unique Protocol Identification Number
NCT00122317
Brief Title
Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official Title
Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH
Study Type
Interventional

2. Study Status

Record Verification Date
February 2018
Overall Recruitment Status
Completed
Study Start Date
May 2005 (Actual)
Primary Completion Date
September 2008 (Actual)
Study Completion Date
October 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Hemoglobinuria, Nocturnal
Keywords
transfusion dependent, paroxysmal nocturnal hemoglobinuria, hemolytic

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
187 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Eculizumab
Arm Type
Experimental
Arm Description
600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Intervention Type
Drug
Intervention Name(s)
eculizumab
Other Intervention Name(s)
Soliris
Primary Outcome Measure Information:
Title
Incidence of Treatment-emergent Adverse Events
Time Frame
From time of consent to a maximum of 2.5 years of study treatment
Secondary Outcome Measure Information:
Title
Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve
Time Frame
From time of first infusion through 24 months of study treatment
Title
Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline
Description
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.
Time Frame
From time of first infusion through 24 months of study treatment
Title
Incidence of Thrombosis After Eculizumab Infusion
Description
Thrombosis was defined as occurrence of major adverse vascular events
Time Frame
From time of first ever dose through last dose (up to 24 months of study treatment)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
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, Department of Clinical Research
City
Weston
State/Province
Florida
ZIP/Postal Code
33331
Country
United States
Facility Name
Indianapolis University Cancer Center
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, Divison of Hematology
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55902
Country
United States
Facility Name
Washington University, Department of Internal Medicine/Division of Hematology
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
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
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thombosis Program
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Royal North Shore Hospital, Haematology Department
City
Saint Leonards
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, Department of Haematology/Level 2
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6000
Country
Australia
Facility Name
Royal Melbourne Hospital, Department 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
N6A4L6
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 Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm
City
Ulm
ZIP/Postal Code
D-89081
Country
Germany
Facility Name
St. James's Hospital, 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, Department 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
State/Province
GA
ZIP/Postal Code
6525
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
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
SW17OQT
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
17702897
Citation
Hillmen P, Muus P, Duhrsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socie G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. doi: 10.1182/blood-2007-06-095646. Epub 2007 Aug 16.
Results Reference
derived

Learn more about this trial

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

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