Cerebellar Ataxia
Conditions
Keywords
Stem Cells, Adult Stem Cells, Spinocerebellar Ataxia, Steminent
Brief summary
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.
Interventions
BIOLOGICALStemchymal®
Patients will receive Stemchymal® through intravenous infusion
PROCEDUREExcipients
Patients will receive excipients through intravenous infusion
Sponsors
Steminent Biotherapeutics Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
20 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Subjects are with genotypically confirmed spinocerebellar ataxia type 2 or spinocerebellar ataxia type 3. 2. Subjects' baseline SARA score are in the range of 8 to15. 3. Subjects are between 20 and 70 years of age. 4. Subjects who had signed informed consent.
Exclusion criteria
1. Subjects had been enrolled in any kind of cell therapy within six months prior to screening visit. 2. Females with a positive pregnancy test result. 3. 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. 4. Subjects with immunological disorders (ex. Systemic lupus erythematosus), within six months prior to screening visit. 5. Subjects with other neurological disorders (ex. Alzheimer's disease), within six months prior to screening visit. 6. Subjects who had received chemotherapy/radiotherapy within five years prior to screening visit. 7. Subjects with any history of malignancy tumors. 8. Subjects with a history of hypersensitivity/allergy to penicillin. 9. Subjects with dementia or other psychiatric illnesses, including but not limited to disabling depression, bipolar disorder, and schizophrenia. 10. Subjects with Beck Depression Inventory Second Edition (BDI-II) score over 20 points. 11. Subjects with unstable illnesses or contraindication for this clinical trial according to PI's judgment.
Design outcomes
Primary
| Measure | 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
| Measure | Time frame | Description |
|---|---|---|
| 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) | 14 months | Balance test |
| 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 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 | — |
| To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS) | 14 months | — |
Countries
Taiwan
Outcome results
None listed