Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia

NCT ID: NCT02540655

Last Updated: 2022-10-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 56 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-09-30
• Study Completion Date: 2021-02-26

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.

Conditions

Cerebellar Ataxia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Stemchymal®

Infusion of Stemchymal®

Group Type: EXPERIMENTAL

Stemchymal®

Intervention Type: BIOLOGICAL

Patients will receive Stemchymal® through intravenous infusion

Vehicle

Infusion of excipients

Group Type: PLACEBO_COMPARATOR

Excipients

Intervention Type: PROCEDURE

Patients will receive excipients through intravenous infusion

Interventions

Stemchymal®

Patients will receive Stemchymal® through intravenous infusion

Intervention Type: BIOLOGICAL

Excipients

Patients will receive excipients through intravenous infusion

Intervention Type: PROCEDURE

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.

• Minimum Eligible Age: 20 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Steminent Biotherapeutics Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Taipei Veterans General Hospital

Taipei, , Taiwan

Countries

Taiwan

Other Identifiers

IB02

Identifier Type: -

Identifier Source: org_study_id