Ataxia UK and CureDRPLA are coordinating the DRPLA Natural History and Biomarker Study for this very rare form of ataxia. The funding for the study is provided by CureDRPLA. This is a global study, including a UK site at the London Ataxia Centre, which is now recruiting participants.
The DRPLA Natural History and Biomarkers Study has three main objectives:
- Researchers would like to characterize the natural history of DRPLA, that is to say, how subjects with DRPLA change over time.
- This study will identify genetic factors and biomarkers that could predict disease progression. A biomarker is any variable/characteristic that informs of physiological processes, pathological processes, or response/effects of a drug.
- This study will provide useful information to support the design and conduct of clinical trials in the future.
This study will collect health information from individuals with DRPLA over three years to understand how this condition develops over time. The study also recruits participants without ataxia, so that they are able to compare how individuals with DRPLA differ from those without. Participating in this study will involve annual appointments at the clinics where the neurologist will do some clinical assessments (e.g. ataxia scales, brain MRI, blood collection, etc.). Find out more about this study in this recruitment flyer.
There are study sites in the United Kingdom (London), the United States (New York and North Carolina) and Japan. Although participants from anywhere in the world could also be recruited.
If you are based in the UK and would like to participate or receive more information, please contact:
Dr Hector Garcia-Moreno and Ms Ola Volhin
UCL Queen Square Institute of Neurology, London WC1N 3BG