INSPIRE-AT Study Final V1.0

  • Research type

    Research Study

  • Full title

    INSPIRE AT: Longitudinal Study Comparing the Natural History of Ataxia Telangiectasia in Children to Healthy Comparators



  • Contact name

    Mohnish Suri

  • Contact email /

  • Sponsor organisation

    Insmed Incorporated

  • Duration of Study in the UK

    2 years, 9 months, 30 days

  • Research summary

    The purpose of this study is to collect data on children with AT as well as Healthy participants to help with future study design and treatment for patients with AT.
    The study aims to evaluate:
    • The symptoms of AT and how they do or do not change over time, by collecting different measurements and disease rating scales
    • The changes with time in the health-related day-to-day life of children with AT
    • Changes in physical activity over time, by utilising an electronic wearable device (an ‘accelerometer’ which is a watch-like device that measures the participant’s movement) and target tracking test (Hevelius test, which is a computer-based test in which the participant is asked to follow a target on screen), and compare the assessment results among different age groups of children with AT, and between healthy children and children with AT
    • Identification of biomarkers (biomarkers are characteristics that are measured and evaluated as a sign of normal or abnormal process, or of a condition or disease, for eg., cholesterol levels, levels of vitamin D, levels of different proteins, etc.) related to AT and compare the levels of these biomarkers between healthy children and children with AT
    No drugs are being given as part of this study. The aim is to measure AT disease symptoms over time to get a better understanding of the disease. It is hoped that in the future this will be useful if and when clinical studies of possible AT drugs are performed.

  • REC name

    Yorkshire & The Humber - Bradford Leeds Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    21 Dec 2023

  • REC opinion

    Favourable Opinion