Clinical trial NCT02540655: Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cerebellar Ataxia
Keywords: Stem Cells
Adult Stem Cells
Spinocerebellar Ataxia
Steminent
Lead sponsor: Steminent Biotherapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2015-09-04
Enrollment: 56
Sex: ALL
Minimum age (years): 20
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.
timeFrame: 14 months
Secondary outcomes:
measure: 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).
timeFrame: 14 months
measure: To assess the changes from the baseline to all visits on SCA functional index (SCAFI)
timeFrame: 14 months
measure: To assess the changes from the baseline to all visits on sensory organization test (SOT)
timeFrame: 14 months
measure: To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)
timeFrame: 14 months
measure: To assess the changes from the baseline to all visits on clinical global impression (CGI)
timeFrame: 14 months
measure: To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)
timeFrame: 14 months
Other outcomes:
Locations:
city: Taipei
country: Taiwan
facility: Taipei Veterans General Hospital
Study officials:
References: