Clinical Trial: A Study to Evaluate the Clinical Pharmacology and Safety of C1-esterase Inhibitor Administered by the Subcutaneous Route

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

Official Title: An Open-label, Cross-over, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of the Subcutaneous Administration of a Human Plasma-derived C1-esterase Inhibitor in Subjec

Brief Summary: The aim of the study is to assess what happens to C1-esterase inhibitor that is administered under the skin of subjects with hereditary angioedema. Three different dosing regimens of C1-esterase inhibitor will be assessed. Each subject will be assigned to receive 2 of the 3 dosing regimens, each for 4 weeks. The activity and concentration of C1-esterase inhibitor in the blood will be measured during each 4-week period. The study will also examine how well C1-esterase inhibitor administered under the skin is tolerated by the subjects.

Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome: Modeled C1-esterase inhibitor functional activity trough level [ Time Frame: 13 time points during each 4-week dose regimen ]

Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens, based on modeling and simulation


Original Primary Outcome: Modeled C1-esterase inhibitor functional activity trough level [ Time Frame: During the 4th week of each dose regimen ]

Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens, based on modeling and simulation


Current Secondary Outcome:

  • C1-esterase inhibitor functional activity trough level [ Time Frame: 2 time points during the last week of each 4-week dose regimen ]
    Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens
  • C1-esterase inhibitor concentration trough level [ Time Frame: 2 time points during the last week of each 4-week dose regimen ]
    Mean trough C1-esterase inhibitor concentration of the low, medium and high subcutaneous dose regimens
  • C4 concentration trough level [ Time Frame: 2 time points during the last week of each 4-week dose regimen ]
    Mean trough C4 concentration of the low, medium and high subcutaneous dose regimens
  • Change in C1-esterase inhibitor functional activity [ Time Frame: After four weeks of treatment with each dose regimen ]
    Mean change from baseline of C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens
  • Change in C1-esterase inhibitor concentration [ Time Frame: After four weeks of treatment with each dose regimen ]
    Mean change from baseline of C1-esterase inhibitor concentration of the low, medium and high subcutaneous dose regimens
  • Change in C4 concentration [ Time Frame: After four weeks of treatment with each dose regimen ]
    Mean change from baseline of C4 concentration of the low, medium and high subcutaneous dose regimens


Original Secondary Outcome:

  • C1-esterase inhibitor functional activity trough level [ Time Frame: During the 4th week of each dose regimen ]
    Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens
  • C1-esterase inhibitor concentration trough level [ Time Frame: During the 4th week of each dose regimen ]
    Mean trough C1-esterase inhibitor concentration of the low, medium and high subcutaneous dose regimens
  • C4 concentration trough level [ Time Frame: During the 4th week of each dose regimen ]
    Mean trough C4 concentration of the low, medium and high subcutaneous dose regimens
  • Change in C1-esterase inhibitor functional activity [ Time Frame: After four weeks of treatment with each dose regimen ]
    Mean change from baseline of C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens
  • Change in C1-esterase inhibitor concentration [ Time Frame: After four weeks of treatment with each dose regimen ]
    Mean change from baseline of C1-esterase inhibitor concentration of the low, medium and high subcutaneous dose regimens
  • Change in C4 concentration [ Time Frame: After four weeks of treatment with each dose regimen ]
    Mean change from baseline of C4 concentration of the low, medium and high subcutaneous dose regimens


Information By: CSL Behring

Dates:
Date Received: April 10, 2012
Date Started: April 2012
Date Completion:
Last Updated: October 5, 2015
Last Verified: June 2014