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

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

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

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
Please refer to this study by its identifier: NCT00122330

  Show 43 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Additional publications automatically indexed to this study by Identifier (NCT Number): 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, Paroxysmal
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases processed this record on April 23, 2014