Clinical Trial: Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase 3, Randomized, Double-blind, Placebo-controlled,Two-period, Three-sequence, Partial Crossover Study to Evaluate the Efficacy and Safety of Subcutaneous Administration of C1 Esterase Inhibitor

Brief Summary: The purpose of this study is to assess the efficacy and safety of subcutaneous administration of a liquid formulation of C1 esterase inhibitor for the prevention of angioedema attacks in adolescent and adult subjects with hereditary angioedema.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome: Time-Normalized Number of Attacks (NNA) for participants [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Proportion of subjects meeting at least a 50% reduction in the NNA during the experimental injection treatment period relative to the placebo period. [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  • Cumulative attack severity [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  • Number of attack-free days [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  • Number of angioedema attacks requiring acute treatment [ Time Frame: Each treatment period: Weeks 1 to 14 ]
  • Response to icatibant when administered for an acute attack [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  • Number of subjects with adverse events (AEs) [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  • Number of patients with injection site reactions [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]


Original Secondary Outcome: Same as current

Information By: Shire

Dates:
Date Received: October 20, 2015
Date Started: January 2016
Date Completion: July 2017
Last Updated: January 13, 2017
Last Verified: January 2017