OPTIMISTIC
Research type
Research Study
Full title
Observational Prolonged Trial in Myotonic Dystrophy type 1 to Improve QoL Standards, a Target Identification Collaboration
IRAS ID
137613
Contact name
Grainne Gorman
Contact email
Sponsor organisation
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Research summary
Myotonic dystrophy type1 (DM1) is a rare, inherited, chronic progressive disease as well as an autosomal dominant multisystemic disorder. It is the most common adult form of muscular dystrophy, with a prevalence of approximately 10 per 100,000 people affected (Norwood 2009, Norman 1989). With 733 million people in Europe, we estimate that 75,000 people are DM1 patients in Europe (European Commission 2011).
OPTIMISTIC is a multi-centre, randomised controlled trial designed to compare a two component tailored behavioural change intervention to increase physical activity against standard patient management regimes, with particular attention given to the definition of appropriate outcome measures and new clinical guidelines for DM1 management. The two components of the intervention are 1) cognitive behavioural therapy (CBT) and 2) graded physical activity and we will evaluate the intervention’s effectiveness and safety against standard patient management.
Participants will be recruited from myotonic dystrophy clinics and neuromuscular centres in the UK, the Netherlands, Germany and France. A total of 286 male and female patients aged 18 years and older with genetically proven classical or adult DM1 suffering from severe fatigue (CIS subscale fatigue score > 35), able to walk independently and able to complete the trial interventions will be included.
In addition, OPTIMISTIC will identify genetic factors that predict outcome and potential biomarkers as surrogate outcome measures that best explain the observed clinical variation.
The intervention will also involve caregivers where they are willing to take part and with the consent of the participant. Caregivers will be asked to complete 3 questionnaires at each of the study visits. If the participant is randomised to the intervention are, the caregivers will also be involved in the CBT sessions.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
13/NE/0342
Date of REC Opinion
20 Dec 2013
REC opinion
Further Information Favourable Opinion