Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

Study Overview
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Brief Summary
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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.

Official Title
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A Phase II, Randomized, Double-Blind, Placebo-Controlled, Single-Center Study to Evaluate the Safety and Efficacy of Stemchymal® Infusion for the Treatment of Polyglutamine Spinocerebellar Ataxia

Conditions
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Cerebellar Ataxia

Intervention / Treatment
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* Biological: Stemchymal®
* Procedure: Excipients


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects are with genotypically confirmed spinocerebellar ataxia type 2 or spinocerebellar ataxia type 3. Subjects' baseline SARA score are in the range of 8 to15. Subjects are between 20 and 70 years of age. Subjects who had signed informed consent. Exclusion Criteria: Subjects had been enrolled in any kind of cell therapy within six months prior to screening visit. Females with a positive pregnancy test result. Subjects who had had severe vital organ disease as diagnosed, including but not limited to cardiac (ex. heart failure), liver (ex. acute hepatic failure or chronic liver cirrhosis), lung (ex. respiratory failure) and renal (ex. hemodialysis or peritoneal dialysis) insufficiency, within six months prior to screening visit. Subjects with immunological disorders (ex. Systemic lupus erythematosus), within six months prior to screening visit. Subjects with other neurological disorders (ex. Alzheimer's disease), within six months prior to screening visit. Subjects who had received chemotherapy/radiotherapy within five years prior to screening visit. Subjects with any history of malignancy tumors. Subjects with a history of hypersensitivity/allergy to penicillin. Subjects with dementia or other psychiatric illnesses, including but not limited to disabling depression, bipolar disorder, and schizophrenia. Subjects with Beck Depression Inventory Second Edition (BDI-II) score over 20 points. Subjects with unstable illnesses or contraindication for this clinical trial according to PI's judgment.

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Stemchymal®<br>Infusion of Stemchymal® | Biological: Stemchymal®<br>* Patients will receive Stemchymal® through intravenous infusion<br>|
| Placebo Comparator: Vehicle<br>Infusion of excipients | Procedure: Excipients<br>* Patients will receive excipients through intravenous infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score. |  | 14 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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). |  | 14 months | 
| To assess the changes from the baseline to all visits on SCA functional index (SCAFI) |  | 14 months | 
| To assess the changes from the baseline to all visits on sensory organization test (SOT) | Balance test | 14 months | 
| To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS) |  | 14 months | 
| To assess the changes from the baseline to all visits on clinical global impression (CGI) |  | 14 months | 
| To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I) |  | 14 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Stem Cells, Adult Stem Cells, Spinocerebellar Ataxia, Steminent