Clinical Trial: A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

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

Official Title: A Double-blind, Randomized, Placebo-controlled, Cross-over Study to Evaluate the Clinical Efficacy and Safety of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophy

Brief Summary: The aim of this study is to assess the efficacy of C1-esterase inhibitor in preventing hereditary angioedema attacks when it is administered under the skin of subjects with hereditary angioedema. The safety of C1-esterase inhibitor will also be assessed. Each subject will enter a run-in period of up to 8-weeks. Subjects who complete the run-in period and who are eligible will then enter the treatment phase which comprises two sequential treatment periods. In the treatment phase, subjects will be randomized to one of four arms consisting of treatment with low- or higher-volume C1-esterase inhibitor in one treatment period and treatment with low- or higher-volume placebo in the other treatment period. The study will measure the number of hereditary angioedema attacks that subjects experience while receiving each treatment.

Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome: The time-normalized number of hereditary angioedema attacks [ Time Frame: During the treatment phase, up to 32 weeks. ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Percentage of subjects with a ≥ 50% reduction in the number of hereditary angioedema attacks [ Time Frame: During the treatment phase, up to 32 weeks. ]
  The percentage of subjects with a ≥ 50% relative reduction in the time-normalized number of hereditary angioedema attacks during treatment with C1-esterase inhibitor compared with placebo
• Number of uses of rescue medication [ Time Frame: During the treatment phase, up to 32 weeks. ]
  The time-normalized number of uses of rescue medication during treatment with C1-esterase inhibitor or placebo
• Percentage of subjects with adverse events (AEs). [ Time Frame: Within 24 hours of C1-esterase inhibitor or placebo administration. ]
• Percentage of subjects with AEs or other specified safety events. [ Time Frame: During the treatment phase, up to 32 weeks. ]
  The percentage of subjects with unsolicited AEs, serious AEs, or other specified safety events during treatment with C1-esterase inhibitor or placebo.
• Percentage of subjects experiencing solicited AEs [ Time Frame: During the treatment phase, up to 32 weeks. ]
  The percentage of subjects experiencing solicited AEs (injection site reactions) during treatment with C1-esterase inhibitor or placebo.
• Percentage of investigational product injections resulting in solicited AEs [ Time Frame: During the treatment phase, up to 32 weeks. ]
  The percentage of injections of C1-esterase inhibitor or placebo that result in solicited AEs (injection site reactions).

Original Secondary Outcome:

• Proportion of subjects with a 50% reduction in the number of hereditary angioedema attacks [ Time Frame: During the treatment phase, up to 32 weeks. ]
  The percentage of subjects with a 50% relative reduction in the time-normalized number of hereditary angioedema attacks during treatment with C1-esterase inhibitor compared with placebo
• Number of uses of rescue medication [ Time Frame: During the treatment phase, up to 32 weeks. ]
  The time-normalized number of uses of rescue medication during treatment with C1-esterase inhibitor or placebo
• Proportion of subjects with any adverse events [ Time Frame: During or within 24 hours of C1-esterase or placebo administration. ]
  The percentage of subjects with any adverse events that begin within 24 hours of administration of C1-esterase inhibitor or placebo

Information By: CSL Behring

Dates:
Date Received: July 29, 2013
Date Started: January 2014
Date Completion:
Last Updated: April 25, 2017
Last Verified: November 2015