Observational Prolonged Trial in Myotonic Dystrophy Type 1 (OPTIMISTIC)
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. With 733 million people in Europe, we estimate that 75,000 people are DM1 patients in Europe.
The aim of OPTIMISTIC is to improve clinical practice in the management of patients with this rare disease for which no dedicated treatment is currently available. 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 France, Germany, the Netherlands and the UK. A total of 286 male and female patients aged 18 years and older with genetically proven classical or adult DM1 suffering from severe fatigue (only DM1 patients with a CIS subscale fatigue score > 35 are likely to benefit from the intervention), able to walk independently and able to complete the trial interventions will be included.
A key objective of OPTIMISTIC is to provide outcome measures that are relevant for the patients and have a rate of change that is appropriate for a clinical trial timeframe. In addition, OPTIMISTIC will identify genetic factors that predict outcome and potential biomarkers as surrogate outcome measures that best explain the observed clinical variation.
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Observational Prolonged Trial in Myotonic Dystrophy Type 1 to Improve Quality of Life Standards, a Target Identification Collaboration|
- DM1-Activ [ Time Frame: Baseline and 10 months ]The primary outcome measure will be the change in DM1-Activ score. DM1-Activ is a specific outcome measure of activity and participation for patients with DM1.
- Six Minute Walk Test [ Time Frame: Baseline and 10 months ]Six minute walk test with BORG scale assessment
- Myotonic Dystrophy Health Index (MDHI) [ Time Frame: Baseline and 10 months ]
- Physical activity measured with actometer [ Time Frame: Baseline and 10 months ]
- Fatigue and Daytime Sleepiness Scale (FDSS) [ Time Frame: Baseline and 10 months ]
- Checklist Individual Strength (CIS) [ Time Frame: Baseline and 10 months ]
- Individualised Neuromuscular Quality of Life Questionnaire (InQoL) [ Time Frame: Baseline and 10 months ]
- Beck depression Inventory for Primary Care [ Time Frame: Baseline and 10 months ]
- Apathy Evaluation Scale (AES) [ Time Frame: Baseline and 10 months ]
- Stroop Test [ Time Frame: Baseline and 10 months ]
- Explanatory and/or Predictive outcomes [ Time Frame: Baseline, 10 and 16 months ]Biomarkers (urine and blood)
|Study Start Date:||April 2014|
|Estimated Study Completion Date:||December 2016|
|Estimated Primary Completion Date:||March 2016 (Final data collection date for primary outcome measure)|
Experimental: Behavioural change intervention
Cognitive behavioural therapy (CBT) combined with exercise
Behavioral: Behavioural change intervention
The intervention is cognitive behaviour therapy (CBT). The CBT consists of six different modules. All patients will start with individual goal setting and psycho-education about the role of cognitive-behavioural variables in the disabilities patients' experience. The patient formulates his or her treatment goals in concrete terms and later on in the therapy the goals are realised step by step by the patient. The treatment is tailored to the patient's problems: which of the six modules a patient will receive is dependent on the scores on measures that have been collected at baseline assessment. Based on our previous experience with modular interventions we expect that most patients will receive less than four modules.
No Intervention: Standard Patient Management
Standard care like usual (i.e. annual checks with neurologist, checks with cardiologist, if needed physical therapy)
Show Detailed Description
Please refer to this study by its ClinicalTrials.gov identifier: NCT02118779
|Assistance Publique-Hospitaux de Paris|
|Friedrich Naur Institute|
|Radboud University Nijmegen Medical Centre|
|Newcastle, United Kingdom, NE1 3BZ|
|Principal Investigator:||Grainne Gorman, Dr||Newcastle University|
|Principal Investigator:||Baziel van Engelen, Prof||Radboud University Nijmegen Medical Centre, The Netherlands|
|Principal Investigator:||Benedikt Schoser, Prof||Munich University, Germany|
|Principal Investigator:||Guillaume Bassez, Prof||Assistance Publique-Hospitaux de Paris, France|