Cinryze in Prevention of Angioedema Attacks in Children

  • Research type

    Research Study

  • Full title

    A phase 3, multicenter, randomized, single-blind, dose-ranging, crossover study to evaluate the safety and efficacy of intravenous administration of Cinryze® (c1 esterase inhibitor [human]) for the prevention of angioedema attacks in children 6 to 11 years of age with hereditary angioedema

  • IRAS ID

    144878

  • Contact name

    Stephen Hughes

  • Contact email

    stephen.hughes@cmft.nhs.uk

  • Sponsor organisation

    ViroPharma Incorporated

  • Eudract number

    2013-002453-29

  • Research summary

    Hereditary Angioedema (HAE) is a disease in which people either do not have enough of a key protein in the blood that protects the body from swelling, or the protein they do have does not work properly. This protein is called C1 inhibitor (C1 INH). People with HAE can develop rapid swelling of the hands, feet, arms, legs, face, abdomen, genital area, or throat/airways. Pain, nausea and vomiting can also occur when there is swelling within the abdomen. These swelling episodes are referred to as angioedema or HAE attacks.
    CINRYZE is approved by the United States Food and Drug Administration (FDA, the Agency that regulates the approval and use of drugs in the United States) to prevent HAE attacks in adolescents and adults. Participants in this study are aged between 6 to 11 years. CINRYZE has not been approved for use in children under the age of 12, so this is why the use of CINRYZE in this study is considered experimental.
    There are three different periods of time which are part of this study. The first period is a baseline observation period, the second is treatment period 1 and the third is treatment period 2.
    The participant will be in the study for about 40 weeks and have approximately 50 study visits, some of which will be at the participant’s home. Study procedures include physical examinations and taking of blood samples. The participant will have an electronic diary to record symptoms and attacks on an ongoing basis throughout the study.
    A total of 12 children with HAE will take part in this study in various countries of the world, including the United States, and countries in the European Union and Latin America.

  • REC name

    North West - Liverpool Central Research Ethics Committee

  • REC reference

    14/NW/0018

  • Date of REC Opinion

    5 Mar 2014

  • REC opinion

    Further Information Favourable Opinion