With no specific treatment for SCA, this five-year clinical trial readiness grant will establish the world’s largest study participant group so that researchers can collect enough clinical, imaging and biochemical data to design full-scale clinical trials that generate conclusive results on effective treatments. The implications of this readiness study are significant, given that there has been no pharmaceutical treatment option approved in the last 25 years for ataxia in the US and Europe.
About SCA1 and SCA3
Spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3) are two of the most common SCAs with clinical characteristics shown below:
SCA1
- Fastest progressing
- Shorter clinical trial timelines due to swift progression
- Sign/symptom onset typically occurs in early adulthood, but can occur between childhood and late adulthood
- Patients eventually require wheelchair assistance
- Survival 10-20 years after first symptoms
SCA3
- Most common SCA in US and Europe, where the study sites are located
- Opportunity for larger number of study participants for clinical trial
- Onset of signs/symptoms typically occurs in mid-adulthood, but can occur between childhood and late adulthood
- Patients eventually require wheelchair assistance
Aims
This clinical trial readiness grant has three specific aims, with different milestones within each aim that occur over the five-year grant period. The three specific aims are: