The full title of OPTIMISTIC is:
"OPTIMISTIC: Observational Prolonged Trial In Myotonic dystrophy type 1 to Improve Quality of Life- Standards, a Target Identification Collaboration."
OPTIMISTIC is a collaboration between researchers and doctors from the Netherlands, Germany, France and the United Kingdom. OPTIMISTIC aims to improve clinical practice and standards of care for patients with DM1 internationally. DM1 is a complex disease affecting an estimated 1 in 8,000 people worldwide. DM1 has often been a neglected disease, with progress in the development of management and therapies lacking behind that of other conditions. OPTIMISTIC represents a change in attitudes and is the first project focusing on DM1 to involve an international multicentre intervention.
The OPTIMISTIC trial aims to:
- Use exercise therapy to improve or at least maintain functional capacity, improve muscle function and increase quality of life.
- Use Cognitive Behavioural Therapy (CBT) to stimulate an active lifestyle, reduce fatigue and increase quality of life.
- Compare the effects of CBT and exercise therapy.
- Develop guidelines for doctors to use to help patients with myotonic dystrophy.
- Improve the quality of life of patients living with DM1.
Other aims of OPTIMISTIC include:
- Validating outcome measures that can be used in clinical trials in the future.
- Developing new techniques for cardiac screening in DM1.
- Using genetic analysis to understand more about what causes DM1 and why it affects people differently.
- Identify and validate biomarkers and mediating factors of the treatment efficacy that can help in future treatment and research.