Clinical Trial: CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

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

Official Title: Open-Label, Single-Dose Study to Evaluate the Response and Pharmacokinetics/Pharmacodynamics of Different Doses of CINRYZE® [C1 Inhibitor (Human)] For Treatment of Acute Angioedema Attacks in Chi

Brief Summary: The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with hereditary angioedema (HAE); and (3) to determine the safety and tolerability following IV administration of CINRYZE in this study population.

Detailed Summary: Each subject received CINRYZE for treatment of a single acute angioedema attack.
Sponsor: Shire

Current Primary Outcome: Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom [ Time Frame: Within 4 hours following treatment ]

Original Primary Outcome: Presence of unequivocal beginning of relief of the defining symptom. [ Time Frame: Within 4 hours following treatment ]

Current Secondary Outcome:

  • Time to Unequivocal Beginning of Relief of the Defining Attack Symptom [ Time Frame: Within 4 hours following treatment ]
  • Time to Complete Resolution of the Attack [ Time Frame: Within 1 week following treatment ]
  • Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations [ Time Frame: Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8 ]
    Data was not reported due to change in planned analysis.


Original Secondary Outcome:

  • Time to unequivocal beginning of relief of the defining symptom. [ Time Frame: Within 4 hours following treatment ]
  • Time to complete resolution of the attack. [ Time Frame: Within 1 week following treatment ]
  • C1INH Levels [ Time Frame: pre-infusion to 1 and 24 hours post-infusion ]


Information By: Shire

Dates:
Date Received: March 24, 2010
Date Started: March 2010
Date Completion:
Last Updated: July 14, 2015
Last Verified: June 2014