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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00122317
First received: July 20, 2005
Last updated: April 24, 2017
Last verified: April 2017
  Purpose
The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.

Condition Intervention Phase
Paroxysmal Hemoglobinuria, Nocturnal Drug: eculizumab Phase 3

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Incidence of Treatment-emergent Adverse Events [ Time Frame: time of consent through 30 days after last study drug dose ]

Secondary Outcome Measures:
  • Incidence of Thrombosis [ Time Frame: from first infusion to last infusion in this study ]
  • Hemolysis as Measured by LDH Area Under the Curve [ Time Frame: from first infusion to last infusion in this study ]
  • Quality of Life as Measured by FACIT-F SCALE [ Time Frame: from first infusion to last infusion in this study ]

Enrollment: 187
Actual Study Start Date: May 2005
Study Completion Date: October 2008
Primary Completion Date: September 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eculizumab Drug: eculizumab
600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Other Name: Soliris

  Eligibility

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00122317

  Show 40 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

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
Study First Received: July 20, 2005
Results First Received: April 24, 2017
Last Updated: April 24, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Alexion Pharmaceuticals:
Transfusion dependent, hemolytic paroxysmal nocturnal hemoglobinuria

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

ClinicalTrials.gov processed this record on September 21, 2017