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

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ClinicalTrials.gov Identifier: NCT00122317
Recruitment Status : Completed
First Posted : July 22, 2005
Results First Posted : June 5, 2017
Last Update Posted : March 13, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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

Condition or disease Intervention/treatment Phase
Paroxysmal Hemoglobinuria, Nocturnal Drug: eculizumab Phase 3

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  (Clinical Trial)
Actual Enrollment : 187 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH
Actual Study Start Date : May 2005
Actual Primary Completion Date : September 2008
Actual Study Completion Date : October 2008


Arm Intervention/treatment
Experimental: Eculizumab
600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Drug: eculizumab
Other Name: Soliris



Primary Outcome Measures :
  1. Incidence of Treatment-emergent Adverse Events [ Time Frame: From time of consent to a maximum of 2.5 years of study treatment ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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.

  3. 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



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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00122317


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Sponsors and Collaborators
Alexion Pharmaceuticals

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00122317     History of Changes
Other Study ID Numbers: E05-001
First Posted: July 22, 2005    Key Record Dates
Results First Posted: June 5, 2017
Last Update Posted: March 13, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Alexion Pharmaceuticals:
transfusion dependent
paroxysmal nocturnal hemoglobinuria
hemolytic

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases