Queen Square Centre for Neuromuscular Diseases


Observational Prolonged Trial in Myotonic Dystrophy type 1 (DM1) to Improve Quality of Life Standards, a Target Identification Collaboration

Sponsor The Newcastle upon Tyne Hospitals NHS Foundation Trust
Funder EU Seventh Framework Programme
CI Dr Grainne Gorman
Sites Newcastle
Contact details Elizabeth.wood2@newcastle.ac.uk
More information one and two

This is a two-arm, multi-centre, randomised controlled trial in DM1 patients.

Primary objective

To compare a specific behavioural change intervention against standard patient management regimes.

The trial and outcome work should lead to new clinical guidelines for DM1 management. The intervention consists of cognitive behavioural therapy and graded physical activity. Both aim to achieve a more active lifestyle. Outcome measures will be assessed at:

  • baseline
  • 5 months
  • 10 months (the end of the intervention period)
  • 6-months post intervention (i.e. 16 months from baseline)