ACE SMA

  • Research type

    Research Study

  • Full title

    A Monocentric, Prospective and Longitudinal study investigating the feasibility, acceptability, safety and efficacy of an optimized rehabilitation program for treated patients with SMA compared to the current rehabilitation program in United Kingdom: Proof of concept of ACE SMA

  • IRAS ID

    326914

  • Contact name

    Laurent Servais

  • Contact email

    laurent.servais@paediatrics.ox.ac.uk

  • Sponsor organisation

    University of Oxford

  • Clinicaltrials.gov Identifier

    326914, IRAS

  • Duration of Study in the UK

    2 years, 0 months, 31 days

  • Research summary

    The aim of this project is to understand the feasibility, acceptability, safety, and potential benefit of optimising current physiotherapy practices for patients with SMA between the ages of one and ten years old, that have been treated with any of the three approved SMA therapies, post-symptomatically. Rather, Instead of only increasing the amount of hands-on physiotherapy to get closer to models applied in countries such as in Belgium/France/Germany/Italy etc, which would currently not be manageable for care teams in a UK setting, we intend to ensure a rigorous follow-up of the physiotherapy care plan by offering a goal-oriented rehabilitation with more frequent hands-on physiotherapy as well as to provide the most appropriate therapy for each individual patient. In addition, as an exploratory approach, we may also offer access to innovative rehabilitation devices at home.

    If successful in changing the prescribed care regime, this project will help develop our understanding of the importance of the frequent and individualized provision of rehabilitation by physiotherapists (PTs) to patients with SMA, and consequently reduce the burden of care on the families.

  • REC name

    West Midlands - South Birmingham Research Ethics Committee

  • REC reference

    23/WM/0275

  • Date of REC Opinion

    28 Dec 2023

  • REC opinion

    Unfavourable Opinion