Clinical Trial Readiness for SCA1 and SCA3 (READISCA)
READISCA was a longitudinal clinical-trial readiness study. The program was funded by the NIH to establish a large cohort of early-stage and symptom-less individuals with spinocerebellar ataxia type 1 (SCA1) and spinocerebellar ataxia type 3 (SCA3). From this cohort, the researchers aimed to validate imaging biomarkers and adapt developments in analyses of small population trials for future clinical trials for SCAs.
Study Participants
172 individuals with a recent diagnosis of SCA1 or SCA3 and unaffected individuals with a family history of SCA1/SCA3
Enrollment in READISCA overlaps significantly with the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA), which is also banked with BioSEND.
Available Data
CLINICAL: performance, observational, and self-reported assessments of motor, cognitive, and functional staging
IMAGING: structural MRI, diffusion tensor imaging (DTI), MR spectroscopy (MRS), and resting state functional MRI (rs fMRI)
GENETIC: SCA mutation/CAG repeat length
Available Biospecimens
DNA from blood, plasma, and CSF.