Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

Phase 2

Completed

• Conditions: Cerebellar Ataxia
• Interventions: Biological: Stemchymal®; Procedure: Excipients

• Registration Number: NCT02540655
• Lead Sponsor: Steminent Biotherapeutics Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-08-26
• Study Start: 2015-09
• Study First Submitted QC: 2015-09-01
• Study First Posted: 2015-09-04
• Primary Completion: 2021-02-26
• Study Completion: 2021-02-26
• Status Verified: 2022-10
• Last Update Submitted: 2022-10-26
• Last Update Posted: 2022-10-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 56

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Stemchymal®	Stemchymal®	Infusion of Stemchymal®
Vehicle	Excipients	Infusion of excipients

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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
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 SCA functional index (SCAFI)	14 months

Trial Locations

Locations (1)

Taipei Veterans General Hospital; 🇨🇳 Taipei, Taiwan