What are the goals of the study?
The COS2 clinical outcome study is evaluating a large group of dysferlinopathy patients to build upon and validate the findings of the first COS study. The specific goals are to:
- Validate outcome measures defined in the first phase of this study in a novel set of patients.
- Evaluate effectiveness of the best outcome measures over a 6-month timeframe.
- Address shortfalls in patient reported outcomes (PRO).
- Refine characteristics of patient sub groups with varying rates of progression and determine inclusion/exclusion criteria for trial recruitment.
- Gain greater understanding of the transition from ambulant to non-ambulant and optimize outcomes that can be used to assess the non-ambulant population.
- Understand how different assessments and scales relate to each other and identify early indicators of change and predictors of decline.
- Set up a system for long-term (beyond COS) collection of natural history data.
- Increase trial site capacity and readiness.
The outcome measures and information identified in this study are needed for the development and success of future clinical trials. In addition, the information gained in this study will provide a better understanding of the disease, ensure optimal care and treatment, and will lead to new and better therapies.