What are we learning?
The first Clinical Outcome Study (COS) started in September 2012 and ended in March 2018. It initially involved 6 visits over 3 years but was extended to include 4 and 5 year visits for some participants.
Below is a list of stats for this first study:
- Study involved 15 clinical sites around the world
- Over 135 clinicians, physios, study coordinators, nurses, statisticians, MRI technicians, and administrators were involved.
- A total of 1,248 clinic visits were completed
- 209 participants were enrolled and 173 completed a 3-year visit
- 83% retention rate
- Ages of participants ranged from 12-88
- 48% male/52% female
- 75% ambulant/25% non-ambulant (at baseline)
- Age of onset of muscle weakness ranged from 3-60 years of age
The average retention rate across all clinical trials testing potential treatments is 70%, so an 83% retention rate for a non-treatment clinical study is AMAZING! Such a high retention rate demonstrates to industry that the dysferlinopathy community is highly motivated and will stay the course. If future clinical trials for dysferlinopathy follow this rate of retention, they will be poised for success.
Several publications have already come out of the analysis of the COS data and many more are on the way
- Harris E, et al. The Clinical Outcome Study for dysferlinopathy: An international multicenter study. Neurol Genet. 2016 Aug 2(4): e89
- Moore UR, et al. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study J Neurol Neurosurg Psychiatry Published Online First: 29 January 2018. doi: 10.1136/jnnp-2017-317329
- Diaz-Manera J, et al. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry 2018;89:1071-1081.
- Moore UR, et al. Assessment of disease progression in dysferlinopathy – a one year cohort study. Neurology 2018 – Neurology 2019; 92(5): e461-E474