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

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

Official Title: An Open-label, Randomized Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Her

Brief Summary: The aim of this study is to assess the long-term safety of C1-esterase inhibitor (C1-INH) in preventing hereditary angioedema (HAE) attacks when it is administered under the skin of subjects with HAE. The safety of participating subjects will be assessed for up to 54 weeks. The long-term efficacy of C1-INH will also be assessed. Each eligible subject will enter the treatment phase, wherein subjects will be randomized to treatment with either low- or medium-volume C1-INH. Subjects who have an insufficient treatment response during the study will be given an opportunity to undergo a dose increase. The study aims to enroll eligible subjects who completed study CSL830_3001 (NCT01912456). Subjects who did not participate in study CSL830_3001 may also participate, if eligible and if space permits. Subjects from the United States (US) who complete Treatment Period 2 will be allowed to participate in an Extension Period. During the Extension Period participating US subjects will continue to receive treatment with open-label CSL830 for up to an additional 88 weeks.

Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome: The person-time incidence rates of specified safety events [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]

The incidence rates of related serious adverse events (SAEs), AEs leading to premature study discontinuation, thromboembolic events, or other specified safety events during treatment with C1-INH


Original Primary Outcome: The person-time incidence rates of specified safety events [ Time Frame: During the treatment phase, up to 52 weeks. ]

The incidence rates of related serious adverse events (SAEs), AEs leading to premature study discontinuation, thromboembolic events, or other specified safety events during treatment with C1-INH


Current Secondary Outcome:

  • Percentage of subjects with SAEs or other specified safety events. [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
  • Percentage of C1-INH injections resulting in solicited AEs (injection site reactions). [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
  • Percentage of subjects with at least 1 solicited AE (injection site reaction). [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
  • Percentage of subjects who become seropositive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus. [ Time Frame: From baseline through the treatment and extension phases, up to 146 weeks. ]
  • Percentage of subjects who experience < 1 HAE attack per 4-week period. [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
  • Percentage of subjects with a ≥ 50% reduction in the time-normalized number of HAE attacks. [ Time Frame: From baseline through the treatment and extension phases, up to 146 weeks. ]


Original Secondary Outcome:

  • Percentage of subjects with SAEs or other specified safety events. [ Time Frame: During the treatment phase, up to 52 weeks. ]
  • Percentage of C1-INH injections resulting in solicited AEs (injection site reactions). [ Time Frame: During the treatment phase, up to 52 weeks. ]
  • Percentage of subjects with at least 1 solicited AE (injection site reaction). [ Time Frame: During the treatment phase, up to 52 weeks. ]
  • Percentage of subjects who become seropositive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus. [ Time Frame: From baseline through the treatment phase, up to 52 weeks. ]
  • Percentage of subjects who experience < 1 HAE attack per 4-week period. [ Time Frame: During the treatment phase, up to 52 weeks. ]
  • Percentage of subjects with a ≥ 50% reduction in the time-normalized number of HAE attacks. [ Time Frame: From baseline through the treatment phase, up to 52 weeks. ]


Information By: CSL Behring

Dates:
Date Received: December 10, 2014
Date Started: December 2014
Date Completion: September 2017
Last Updated: May 8, 2017
Last Verified: May 2017