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

This study has been completed.
Sponsor:
Information provided by:
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00122317
First received: July 20, 2005
Last updated: January 14, 2009
Last verified: January 2009
  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

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
Endpoint Classification: Safety Study
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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Incidence of thrombosis [ Time Frame: during study up to 16 weeks post study drug dosing ] [ Designated as safety issue: No ]
  • Hemolysis as measured by LDH area under the curve [ Time Frame: during study ] [ Designated as safety issue: No ]
  • Quality of Life as measured by FACIT-F SCALE [ Time Frame: during study ] [ Designated as safety issue: No ]

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
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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

No publications provided by Alexion Pharmaceuticals

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Jing Jing Wang, MD. Associate Medical Director, Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00122317     History of Changes
Other Study ID Numbers: E05-001
Study First Received: July 20, 2005
Last Updated: January 14, 2009
Health Authority: United States: Food and Drug Administration

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

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

ClinicalTrials.gov processed this record on October 29, 2014