HOW TO TAKE ACTION
COS2 – Study Results
HOW TO TAKE ACTION
COS2 – Study Results
Home ❯ Patients/Clinicians ❯ How to Take Action ❯ Clinical Trials ❯ COS2 ❯ Study Results
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!
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.
Current publications: ordered from earliest to most recent
- Harris E, et al. The Clinical Outcome Study for dysferlinopathy: An international multicenter study. Neurol Genet. 2016 Aug 2(4): e89
- 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
- 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
- Moore UR, et al. Intensive teenage activity is associated with greater muscle pathology on T1W magnetic resonance imaging in adults with Dysferlinopathy. Front Neurol. 2020; 11: 613446.
- Moore UR, et al. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease. Neuromuscul Disord. 2021 Jan 21;S0960-8966(21)00010-9.
- Jacobs M, et al. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale. Ann Neurol. 2021 Feb 11. doi: 10.1002/ana.26044.
- Mayhew et al. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach. Front Neurol. 2022; 13:828525.
- Moore U et al. Cardiac and pulmonary findings in dysferlinopathy: A 3-year, longitudinal study. Muscle and Nerve. February 2022.
- Reyngoudt H, et al. Three-year quantitative MRI and 31P MRS study in lower limb muscle in dysferlinopathy. J Cachexia Sarcopenia Muscle. 2022 Jun; 13(3):1850-1863.
- Reash et al. Comparison of strength testing modalities in dysferlinopathy. Muscle and Nerve. 2022 May 4.
- Moore U et al. Water T2 could predict functional decline in patients with dysferlinopathy. J Cachexia Sarcopenia Muscle. 2022 Dec; 13(6):2888-2897.
- Llansó L, et al. Expanding the muscle imaging spectrum in dysferlinopathy: description of an outlier population from the classical MRI pattern. Neuromuscul Disord. 2023 Mar 2; 33(4):349-357.
- Dolano-Diaz C, et al. Magnetic resonance imaging-based criteria to differentiate dysferlinopathy from other genetic muscle diseases. Neuromuscul Disord. 2023 Nov 10:S0960-8966(23)00793-9. doi: 10.1016/j.nmd.2023.11.004
- Borland et al. Performance of upper limb entry item to predict forced vital capacity in dysferlin-deficient limb girdle muscular dystrophy. Neuromuscul Disord. 2024 Aug 14; doi.org/10.1016/j.nmd.2024.08.003