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

February 11, 2018 updated by: Alexion Pharmaceuticals

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

187

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Parkville, Australia, 3050
        • Royal Melbourne Hospital, Department of Clinical Haematology & Medical Oncology
    • New South Wales
      • Saint Leonards, New South Wales, Australia, 2065
        • Royal North Shore Hospital, Haematology Department
    • Queensland
      • Woolloongabba, Queensland, Australia, 4102
        • Princess Alexandra Hospital, Oncology Haematology Radiation Department
    • South Australia
      • Woodville South, South Australia, Australia, 5011
        • The Queen Elizabeth Hospital, Haematology/Oncology Department
    • Western Australia
      • Perth, Western Australia, Australia, 6000
        • The Royal Perth Hospital, Department of Haematology/Level 2
  • Belgium
      • Brussels, Belgium, 1200
        • Ucl St. Luc, Hematology Department
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2B7
        • University of Alberta, Cross Cancer Institute
    • Ontario
      • London, Ontario, Canada, N6A4L6
        • London Regional Cancer Centre, Clinical Research Unit Room C3080
  • France
    • Cedex
      • Paris, Cedex, France, 10 75475
        • Hopital Saint Louis, Centre d'investigation Clinique
  • Germany
      • Essen, Germany, D-45147
        • Universitatsklinikum Essen, Zentrum fur Innere Medizin
      • Hannover, Germany, D-30625
        • Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
      • Homburg/Saar, Germany, D-66421
        • Universitatskliniken des Saarlandes, Innere Medizin 1
      • Ulm, Germany, D-89081
        • Institut fur Klinische Transfusionmedizin und Immunogenetik, Abtlg. Transfusionmedizin des Univ. Ulm
  • Ireland
      • Dublin, Ireland, 8
        • St. James's Hospital, Cancer Clinical Trial Office
  • Italy
      • Firenze, Italy, 50139
        • Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
      • Genova, Italy, I-16132
        • Ospedale San Martino, Department of Hematology
      • Milano, Italy, 35-20122
        • Ospedale Maggiore di Milano, Divisione di Ematologia
      • Napoli, Italy, 5-80131
        • Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
      • Vicenza, Italy, 37-36100
        • Ospedale San Bortolo, Divisione di Ematologia
  • Netherlands
    • GA
      • Nijmegen, GA, Netherlands, 6525
        • UMC St. Radboud, Department of Hematology
  • Spain
      • Barcelona, Spain, 08036
        • Hospital Clinic I Provincial, Servicio de Hematologia
      • Barcelona, Spain, 08916
        • Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
      • Madrid, Spain, 28046
        • Hospital De La Paz, Servicio de Hematologia
  • Sweden
      • Stockholm, Sweden, 118-83
        • Stockholm South Hospital, Division of Hematology
  • United Kingdom
      • Leeds, United Kingdom, LS1 3EX
        • Leeds General Infirmary, D Floor Brotherton Wing
      • London, United Kingdom, SW17OQT
        • St. George's Hospital, Department of Haematology
  • United States
    • California
      • Stanford, California, United States, 94305
        • Stanford University Medical Center, Division of Hematology
    • Connecticut
      • Hartford, Connecticut, United States, 06102
        • Hartford Hospital, Cancer Clinical Research Office
    • Florida
      • Weston, Florida, United States, 33331
        • Cleveland Clinic Florida, Department of Clinical Research
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indianapolis University Cancer Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Johns Hopkins University Medical Center
      • Bethesda, Maryland, United States, 20892
        • National Heart, Lung, and Blood Institute, National Institutes of Health
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55902
        • Mayo Clinic, Divison of Hematology
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University, Department of Internal Medicine/Division of Hematology
    • New York
      • New York, New York, United States, 10016
        • Nyu Clinical Cancer Center
      • New York, New York, United States, 10017
        • Memorial Sloan Kettering Cancer Center
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic Foundation
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thombosis Program

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Eculizumab
600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Drug: eculizumab
Other Names:
  • Soliris

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of Treatment-emergent Adverse Events
Time Frame: From time of consent to a maximum of 2.5 years of study treatment
From time of consent to a maximum of 2.5 years of study treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
From time of first infusion through 24 months of study treatment
Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline
Time Frame: From time of first infusion through 24 months of study treatment
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
Incidence of Thrombosis After Eculizumab Infusion
Time Frame: From time of first ever dose through last dose (up to 24 months of study treatment)
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)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 1, 2005

Primary Completion (Actual)

September 1, 2008

Study Completion (Actual)

October 1, 2008

Study Registration Dates

First Submitted

July 20, 2005

First Submitted That Met QC Criteria

July 20, 2005

First Posted (Estimate)

July 22, 2005

Study Record Updates

Last Update Posted (Actual)

March 13, 2018

Last Update Submitted That Met QC Criteria

February 11, 2018

Last Verified

February 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • E05-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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