Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

Cerebellar Ataxia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02540655
• Other study ID #: IB02
• Status: Completed
• Phase: Phase 2
• Start date: September 2015
• Est. completion date: February 26, 2021

Study information

• Verified date: October 2022
• Source: Steminent Biotherapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 56
• Est. completion date: February 26, 2021
• Est. primary completion date: February 26, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

Biological:
• Stemchymal®
Patients will receive Stemchymal® through intravenous infusion

Procedure:
• Excipients
Patients will receive excipients through intravenous infusion

Locations

Country	Name	City	State
Taiwan	Taipei Veterans General Hospital	Taipei

Sponsors (1)

Lead Sponsor	Collaborator
Steminent Biotherapeutics Inc.

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.		14 months
Secondary	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
Secondary	To assess the changes from the baseline to all visits on SCA functional index (SCAFI)		14 months
Secondary	To assess the changes from the baseline to all visits on sensory organization test (SOT)	Balance test	14 months
Secondary	To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)		14 months
Secondary	To assess the changes from the baseline to all visits on clinical global impression (CGI)		14 months
Secondary	To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)		14 months