Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Study Identifier:
C07-001
ClinicalTrials.gov Identifier:
NCT01192399
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Paroxysmal Nocturnal Hemoglobinuria
Study Drug
  • Biological: Eculizumab
Date
Nov 2007 - Jun 2008
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 12+ years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Inclusion Criteria:
  • Japanese individuals at least 12 years of age
  • Diagnosis of PNH \> 6 months
  • At least one transfusion in the past 2 years for anemia or anemia-related symptoms
  • LDH level ≥ 1.5 x upper limit of normal within 12 weeks
  • Presence of a glycosylphosphatidylinositol (GPI)-deficient red blood cell (RBC) clone (type III cells) by flow cytometry of ≥10%
  • Negative serum pregnancy test for women of child-bearing potential
  • Exclusion Criteria:
  • Platelet count \< 30,000/µL
  • Absolute neutrophil count ≤ 500/µL
  • Known or suspected hereditary complement deficiency
  • History of hematopoietic stem cell transplant
  • History of meningococcal disease
Sex
Female & Male
Age
12+ years

Study Details

Medical Condition
  • Paroxysmal Nocturnal Hemoglobinuria
Study Drug
  • Biological: Eculizumab
Date
Nov 2007 - Jun 2008
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 12+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Inclusion Criteria:
  • Japanese individuals at least 12 years of age
  • Diagnosis of PNH \> 6 months
  • At least one transfusion in the past 2 years for anemia or anemia-related symptoms
  • LDH level ≥ 1.5 x upper limit of normal within 12 weeks
  • Presence of a glycosylphosphatidylinositol (GPI)-deficient red blood cell (RBC) clone (type III cells) by flow cytometry of ≥10%
  • Negative serum pregnancy test for women of child-bearing potential
  • Exclusion Criteria:
  • Platelet count \< 30,000/µL
  • Absolute neutrophil count ≤ 500/µL
  • Known or suspected hereditary complement deficiency
  • History of hematopoietic stem cell transplant
  • History of meningococcal disease

Protocol Summary

A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.

Trial Locations

No locations found.