Outcome measures are the tests used to decide whether a treatment being tested in a trial is having any effect. Using the right outcome measure is vital to making sure a trial can accurately assess whether or not a treatment works. They can be a physical test for example how far someone can walk or a biological indicator (biomarker) for example a specific molecule in the blood. The varied and complex nature of the condition increases the challenge in validating outcome measures for DM1.
The main aims of this part of OPTIMISTIC are:
- Assess the effect of cognitive behavioural therapy and exercise in DM1 using appropriate outcome measures.
- Establish and validate outcome measures that can be used for clinical trials in the future.