The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments. Through US-European collaborations, the investigators will establish the world's largest cohorts of subjects at the earliest disease stages, who will benefit most from treatments, validate an ability to detect disease onset and early progression by imaging markers, even prior to ataxia onset, and identify clinical trial designs that will generate the most conclusive results on treatment efficacy with small populations of patients.
Age range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Change in disease progression in SCA1 and SCA3 as determined by change in scale for the assessment and rating of ataxia (SARA) score over time.
Timeframe: Every 12 months for approximately 60 months
Change in disease progression in SCA1 and SCA3 as determined by change in Composite Cerebellar Functional Severity Score (CCFS) total score over time.
Timeframe: Every 12 months for approximately 60 months
Change in disease progression in SCA1 and SCA3 as determined by change in timed 25 foot walk test (T25FW) over time.
Timeframe: Every 12 months for approximately 60 months
Change in disease progression in SCA1 and SCA3 as determined by change in Cerebellar Cognitive Affective Syndrome (CCAS) score over time.
Timeframe: Every 12 months for approximately 60 months
Change in disease progression in SCA1 and SCA3 as determined by change in Inventory of Non-ataxia Symptoms (INAS) total count over time.
Timeframe: Every 12 months for approximately 60 months
Change in disease progression in SCA1 and SCA3 as determined by change in Functional staging score over time.
Timeframe: Every 12 months for approximately 60 months
Change in level of disease activity based on change in cerebellar and brainstem volumes since baseline imaging. (Aim 2)
Timeframe: Every 12 months for approximately 36 months
Change in level of disease activity based on grey matter (GM) and white matter (WM) loss metrics from voxel-based morphometric (VBM) since baseline imaging. (Aim 2)
Timeframe: Every 12 months for approximately 36 months
Change in level of disease activity based on change in metabolite concentrations since baseline imaging. (Aim 2)
Timeframe: Every 12 months for approximately 36 months
Change in level of disease activity based on change in fractional isotropy since baseline imaging. (Aim 2)
Timeframe: Every 12 months for approximately 36 months
Change in level of disease activity based on change in mean diffusivity since baseline imaging. (Aim 2)
Timeframe: Every 12 months for approximately 36 months
Change in level of disease activity based on change in radial and axial diffusivity since baseline imaging. (Aim 2)
Timeframe: Every 12 months for approximately 36 months
Change in level of disease activity based on change in degree of co-activation within resting state networks since baseline imaging. (Aim 2)
Timeframe: Every 12 months for approximately 36 months