OPTIMISTIC. Observational prolonged trial in myotonic dystrophy type 1 to improve stamina, a target identification collaboration

Myotonic dystrophy type1 (DM1) is a rare, inherited chronic progressive disease as well as an autosomal dominant multisystemic disorder. It is probably the most common adult form of muscular dystrophy, with a prevalence of approximately 10 per 100,000 people affected.  DM1 has often been a neglected disease, with progress in the development of management and therapies lacking behind that of other conditions. 

OPTIMISTIC is a multi-centre, randomised trial designed to compare an intervention comprising cognitive behavioural therapy plus graded exercise therapy against standard care. Participants will be recruited from myotonic dystrophy clinics and neuromuscular centres in France, Germany, the Netherlands and the UK.  OPTIMISTIC aims to recruit 296 individuals to achieve a sample size of 208 individuals for analysis. 

The project is led by Baziel van Engelen at Radboud University, the Netherlands, is ongoing and recruitment is expected to finish by the middle of 2015.


Shaun Treweek;