We updated the design of this site on December 18, 2017. Learn more.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00122330
Recruitment Status : Completed
First Posted : July 22, 2005
Last Update Posted : December 4, 2006
Sponsor:
Information provided by:

Study Description
Brief Summary:
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.

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

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 75 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Educational/Counseling/Training
Official Title: Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
Study Start Date : October 2004
Estimated Study Completion Date : January 2006


Arms and Interventions


Outcome Measures

Eligibility Criteria

Information from the National Library of Medicine

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, Learn About Clinical Studies.


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

Inclusion Criteria:

  • Must have required at least 4 transfusions in the past 12 months
  • PNH type III red blood cell (RBC) clone by flow cytometry of >10%
  • Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal
  • Platelet count > 100,000/mm3
  • Patient taking erythropoietin must be on a stable dose for at least 26 weeks
  • Patient taking immunosuppressants must be on a stable dose for at least 26 weeks
  • Patient taking corticosteroids must be on a stable dose for at least 4 weeks
  • Patient taking coumadin must be at a stable INR for at least 4 weeks
  • Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks
  • Willing and able to give written informed consent
  • Must avoid conception

Exclusion Criteria:

  • Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl
  • Absolute neutrophil count <500/ul
  • Active bacterial infection
  • Hereditary complement deficiency
  • Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days
  • Pregnant, breast-feeding, or intending to conceive
  • History of meningococcal disease
  • History of bone marrow transplantation
Contacts and Locations

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): NCT00122330


  Show 43 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals