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

This study has been completed.
Information provided by:
Alexion Pharmaceuticals Identifier:
First received: July 20, 2005
Last updated: January 14, 2009
Last verified: January 2009
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

Alexion Pharmaceuticals has indicated that access to an investigational treatment associated with this study is available outside the clinical trial.  

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: 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:
  • Treatment-emergent adverse events [ Time Frame: during study up to 30 days post last study drug dosing ]

Secondary Outcome Measures:
  • Incidence of thrombosis [ Time Frame: during study up to 16 weeks post study drug dosing ]
  • Hemolysis as measured by LDH area under the curve [ Time Frame: during study ]
  • Quality of Life as measured by FACIT-F SCALE [ Time Frame: during study ]

Enrollment: 187
Study Start Date: May 2005
Study Completion Date: October 2008
Primary Completion Date: September 2008 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: eculizumab
    600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
    Drug: eculizumab
    eculizumab 600 mg x 4 every week then eculizumab 900 mg every two weeks

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

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
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Please refer to this study by its identifier: NCT00122317

  Show 40 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Jing Jing Wang, MD. Associate Medical Director, Alexion Pharmaceuticals Identifier: NCT00122317     History of Changes
Other Study ID Numbers: E05-001
Study First Received: July 20, 2005
Last Updated: January 14, 2009

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

Additional relevant MeSH terms:
Hemoglobinuria, Paroxysmal
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms processed this record on May 25, 2017