The purpose of the clinical trial is to study the therapeutic efficacy and safety of Stemchymal® infusions for polyglutamine spinocerebellar ataxia treatment by a randomized, double-blind, placebo-controlled study design. Eligible subjects will receive Stemchymal® through intravenous infusion.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
56
Patients will receive Stemchymal® through intravenous infusion
Patients will receive excipients through intravenous infusion
Taipei Veterans General Hospital
Taipei, Taiwan
To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.
Time frame: 14 months
To assess the safety by incidence of adverse events, changes of vital signs, blood biochemistry tests, complete blood count, immunoactivity assay, urinalysis and magnetic resonance imaging (MRI).
Time frame: 14 months
To assess the changes from the baseline to all visits on SCA functional index (SCAFI)
Time frame: 14 months
To assess the changes from the baseline to all visits on sensory organization test (SOT)
Balance test
Time frame: 14 months
To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)
Time frame: 14 months
To assess the changes from the baseline to all visits on clinical global impression (CGI)
Time frame: 14 months
To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)
Time frame: 14 months
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