Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Study Identifier:
C07-001
ClinicalTrials.gov Identifier:
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.