Facilitating Successful Clinical Trials in the UK

Principal Investigator: Professor Francesco Muntoni

Institution: UCL Institute of Child Health

Funding: £218,903 (split with Muscular Dystrophy UK)

Year awarded: 2016

Duration: 2 years + no-cost extension

The SMA Trust previously funded a 2-year initial project, with the aim of facilitating successful clinical trials in the UK.Research teams at Great Ormond Street Hospital / UCL Institute of Child Health and Newcastle University have now created invaluable tools in preparation for clinical trials in two important ways:

  1. By providing clinicians/researchers with a single database which collects diagnostic data and accurate clinical details on a large collection of SMA patients over a long period, enabling accurate measurement of change
  2. By revising/improving physiotherapy assessment scaled used to assess people living with SMA.  These are now being used nationally and as part of a collaboration with US and Italian networks.

Part 2 of this project involves expanding the network nationally and further validating the assessment scale. Additionally, the team is looking at correlating clinical outcomes to biomarkers, as this could help explain why the disease progresses differently in different patients. Data from children with SMA Type 1 is also now being collected.