Screening Study of Bleeding & Treatment in Participants with VWD

  • Research type

    Research Study

  • Full title

    A Prospective, Screening Study of Bleeding and Treatment in Participants with Von Willebrand Disease

  • IRAS ID

    346002

  • Contact name

    Ulrike Lorch

  • Contact email

    u.lorch@richmondpharmacology.com

  • Sponsor organisation

    Hemab ApS

  • Duration of Study in the UK

    1 years, 6 months, 16 days

  • Research summary

    Von Willebrand disease (VWD) is an inherited bleeding disorder, characterised by bleeding events that range from frequent, low-volume bleeding events, that impact quality of life to large-volume bleeding events that are life-threatening.

    A screening study is being conducted to gather information on bleeding events, quality of life and the social and clinical impact of bleeds in participants with Von Willebrand disease (VWD) to provide a comparison for future clinical studies. The study will also inform the choice of study assessments to be used in future treatment studies and identify potential study participants.

    The study includes screening, a baseline evaluation on Day 1, an observational period of 4-6 months and an optional extension to the observational period of up to 12 months. The study will take approximately 4.5 to 12.5 months to complete (including an optional extension to the observational period) for each participant. The observational period will include phone calls or messages with the study staff to support and monitor the bleed diary and bleeding event treatment record. Participants will be required to have in-person clinic visits every 12 weeks for blood samples to be taken.

  • REC name

    London - Riverside Research Ethics Committee

  • REC reference

    24/LO/0613

  • Date of REC Opinion

    22 Aug 2024

  • REC opinion

    Further Information Favourable Opinion