Clinical Trial: C07-001: Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Brief Summary: The objective of this study was to assess the safety and efficacy of eculizumab in hemolytic PNH patients. In addition, the pharmacokinetics (PK) and pharmacodynamic (PD) response of eculizumab was assessed and compared to overseas studies of eculizumab treatment in hemolytic PNH patients.

Detailed Summary:
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: Change of LDH [ Time Frame: Baseline to Study Week 12 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change of total scores of FACIT-Fatigue scale [ Time Frame: Baseline to Study Week 12 ]
  • Change of PNH RBC count [ Time Frame: Baseline to Study Week 12 ]
  • Change of number of units of Packed RBCs transfused [ Time Frame: Baseline through study period ]
  • AUC for change of LDH [ Time Frame: From baseline ]
  • Change of plasma free hemoglobin [ Time Frame: From baseline to Study Week 12 ]
  • Change of EORTC QLQ-C30 score [ Time Frame: From baseline to Study Week 12 ]


Original Secondary Outcome: Same as current

Information By: Alexion Pharmaceuticals

Dates:
Date Received: August 30, 2010
Date Started: November 2007
Date Completion:
Last Updated: February 15, 2017
Last Verified: February 2017