Researchers from The German Centre for Neurodegenerative Diseases (DZNE) in Tübingen, and the ESMI Consortium, have published a study looking at the potential for gait measurements to quantify progression of SCA3 over time.
When planning an ataxia clinical trial, it is extremely important that researchers have a reliable way to measure the progression of ataxia over time. This allows them to compare ataxia symptoms before and after taking the treatment, to see if the treatment they are testing is effective at preventing or slowing the progression of ataxia. These measurements are called biomarkers.
Gait measurements are under investigation as potential biomarkers for ataxia in a number of studies. Measures of gait have been shown to correlate with ataxia severity in several studies. However, in order to be useful as a biomarker of progression, gait measurements have to be sensitive enough to detect changes over time, ideally within a short time frame (e.g. 1 year).
In this study, researchers measured a number of different features of gait from people with SCA3, and control subjects without ataxia. They were able to measure a change in the features of gait after one year, compared to the measurements taken at the start. The researchers showed that gait measurements were more sensitive to change than clinical ataxia rating scales (such as SARA).
It is key for upcoming clinical trials that researchers can measure changes in the symptoms of ataxia in a way that is sensitive and reliable. This study demonstrated that gait measurements have the potential to be used as an outcome measure in future clinical trials. This was a small study involving 17 people with SCA3. Larger studies are needed to confirm these promising results.
Read the full paper here.