Status:

COMPLETED

Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Lead Sponsor:

Alexion Pharmaceuticals, Inc.

Conditions:

Paroxysmal Nocturnal Hemoglobinuria

Eligibility:

All Genders

12+ years

Phase:

PHASE2

Brief Summary

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

Detailed Description

The objective of this study was to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH. In addition, the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab ...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

November 1 2007

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

June 1 2008

Estimated Enrollment :

29 Patients enrolled

Trial Details

Trial ID

NCT01192399

Start Date

November 1 2007

End Date

June 1 2008

Last Update

March 13 2018

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