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

This study has been completed.
Sponsor:
Information provided by:
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00122330
First received: July 18, 2005
Last updated: November 30, 2006
Last verified: November 2006
  Purpose

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


Condition Intervention Phase
Hemoglobinuria, Paroxysmal
Drug: eculizumab
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Educational/Counseling/Training
Official Title: Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Estimated Enrollment: 75
Study Start Date: October 2004
Estimated Study Completion Date: January 2006
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
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: NCT00122330

  Show 43 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Publications:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
ClinicalTrials.gov Identifier: NCT00122330     History of Changes
Other Study ID Numbers: TRIUMPH
Study First Received: July 18, 2005
Last Updated: November 30, 2006
Health Authority: United States: Food and Drug Administration

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 16, 2014