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

Phase 3

Completed

• Conditions: Hemoglobinuria, Paroxysmal

• Registration Number: NCT00122330
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2004-10
• Study First Submitted: 2005-07-18
• Study First Submitted QC: 2005-07-19
• Study First Posted: 2005-07-22
• Study Completion: 2006-01
• Status Verified: 2006-11
• Last Update Submitted: 2006-11-30
• Last Update Posted: 2006-12-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 75

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Trial Locations

Locations (43)

City of Hope National Medical Center, Div. of Hematology & Bone Marrow Transplant; 🇺🇸 Duarte, California, United States

University of California at Los Angeles; 🇺🇸 Los Angeles, California, United States

Scripps Cancer Center; 🇺🇸 San Diego, California, United States

Stanford University Medical Center, Division of Hematology; 🇺🇸 Stanford, California, United States

Hartford Hospital, Cancer Clinical Research Office; 🇺🇸 Hartford, Connecticut, United States

Cleveland Clinic, Dept. of Clinical Research; 🇺🇸 Weston, Florida, United States

Indiana University Cancer Pavilion, Div. of Hematology-Oncology, Hematological Malignancy Program/Immunology; 🇺🇸 Indianapolis, Indiana, United States

Johns Hopkins University Medical Center; 🇺🇸 Baltimore, Maryland, United States

National Heart, Blood, and Lung Institute, National Institutes of Health; 🇺🇸 Bethesda, Maryland, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

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