Clinical Trial: Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Open-label, Phase II, Single Arm Study to Evaluate the Safety, Immunogenicity, Pharmacokinetics and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Pediatric Patients

Brief Summary: This open-label study is being conducted to confirm the safety, pharmacokinetic profile and efficacy of Ruconest at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in patients, from 2 up to and including 13 years of age.

Detailed Summary:
Sponsor: Pharming Technologies B.V.

Current Primary Outcome:

  • The primary objective is the assessment of safety and tolerability (adverse events, physical examination, vital signs, immunological and routine laboratory analyses et cetera) [ Time Frame: 90 Days ]
  • Immunogenicity by assessing antibodies against recombinant human C1INH (IgG and IgM) anti-rhC1INH) [ Time Frame: 90 Days ]
  • Immunogenicity by assessing antibodies against host related impurities (anti-HRI) [ Time Frame: 90 Days ]
  • Immunogenicity by assessing IgE antibodies against rabbit epithelium [ Time Frame: 28 Days ]


Original Primary Outcome: The primary objective is the assessment of safety and tolerability (adverse events, physical examination, vital signs, immunological and routine laboratory analyses et cetera) [ Time Frame: 90 Days ]

Current Secondary Outcome:

  • Beginning of relief [ Time Frame: 24 Hours ]
    Time to beginning of relief assessed by using the overall severity visual analog scale(VAS), defined as the first time point with a decrease of at least 20 mm with respect to baseline at any eligible location, with persistence at the next time point
  • Minimal symptoms [ Time Frame: 24 Hours ]
    Time to minimal symptoms assessed by using the overall severity VAS, defined as the first time point at which the overall severity VAS falls below 20 mm for each assessed location
  • Complete resolution [ Time Frame: 28 Days (diary recording) ]
    Patient-recorded time at which all angioedema symptoms at all locations have resolved
  • Pharmacokinetic parameters for first attack [ Time Frame: 4 Hours ]
    C1INH activity
  • Pharmacodynamic parameters for first attack [ Time Frame: 4 Hours ]
    C4 levels


Original Secondary Outcome:

  • Immunogenicity by assessing antibodies against recombinant human C1INH (IgG and IgM) anti-rhC1INH) [ Time Frame: 90 Days ]
  • Immunogenicity by assessing antibodies against host related impurities (anti-HRI) [ Time Frame: 90 Days ]
  • Beginning of relief [ Time Frame: 24 Hours ]
    Time to beginning of relief assessed by using the overall severity visual analog scale(VAS), defined as the first time point with a decrease of at least 20 mm with respect to baseline at any eligible location, with persistence at the next time point
  • Minimal symptoms [ Time Frame: 24 Hours ]
    Time to minimal symptoms assessed by using the overall severity VAS, defined as the first time point at which the overall severity VAS falls below 20 mm for each assessed location
  • Complete resolution [ Time Frame: 28 Days (diary recording) ]
    Patient-recorded time at which all angioedema symptoms at all locations have resolved
  • Pharmacokinetic parameters for first attack [ Time Frame: 4 Hours ]
    C1INH activity
  • Pharmacodynamic parameters for first attack [ Time Frame: 4 Hours ]
    C4 levels


Information By: Pharming Technologies B.V.

Dates:
Date Received: May 19, 2011
Date Started: December 2011
Date Completion: December 2017
Last Updated: October 14, 2016
Last Verified: October 2016