Rare and Undiagnosed Diseases Study (RUDY)

  • Research type

    Research Study

  • Full title

    Rare and Undiagnosed Diseases Study (RUDY)

  • IRAS ID

    213780

  • Contact name

    Muhammad K Javaid

  • Contact email

    Kassim.javaid@NDORMS.OX.AC.UK

  • Sponsor organisation

    The University of Oxford

  • Duration of Study in the UK

    5 years, 0 months, 1 days

  • Research summary

    Rare diseases have a significant impact on quality of life and are difficult to diagnose because of their variety and rarity.
    Each has a differing impact on both the patient and the NHS. There is an urgent need to improve the care of these patients by developing novel diagnostic tests and therapies.

    More detailed phenotyping of groups of patients with rare diseases would lead to improved predictive, diagnostic and therapeutic measures.

    This application is to develop a national cohort of participants with rare diseases. From this cohort we will characterize patients using their clinical history, physical examination, blood, urine and genetics to describe different patient groups within each rare diagnosis to understand their disease better. We will then use this understanding to inform novel biomarkers and therapeutic targets.

    Participant and clinician reported study data would be collected via a secure interactive computer based web interface system. There will be paper-based methods of participation in place for those who cannot, or chose not to, access the computer system. Participants will have the opportunity to manage their consent to the project via a dynamic consent system, which allows participants to give varying levels of consent for use of their data and samples, and allows participants to amend these preferences in the future.

  • REC name

    South Central - Berkshire B Research Ethics Committee

  • REC reference

    17/SC/0501

  • Date of REC Opinion

    2 Nov 2017

  • REC opinion

    Further Information Favourable Opinion