Stemchymal® for Polyglutamine Spinocerebellar Ataxia

NCT ID: NCT06397274

Last Updated: 2024-05-02

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-06-01
• Study Completion Date: 2028-06-01

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

Spinocerebellar Ataxias

Study Design

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

Study Groups

Stemchymal®

Group Type: EXPERIMENTAL

Stemchymal

Intervention Type: BIOLOGICAL

Patients will receive Stemchymal® through intravenous infusion

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: BIOLOGICAL

Patients will receive Placebo through intravenous infusion

Interventions

Stemchymal

Patients will receive Stemchymal® through intravenous infusion

Intervention Type: BIOLOGICAL

Placebo

Patients will receive Placebo through intravenous infusion

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. Subjects are with genotypically confirmed SCA3.

2. Subjects' SARA scores are in the range of 5 to15.

3. Subjects are between 20 and 70 years of age.

4. Subjects who have signed informed consent.

5. Female subjects of child-bearing potential who are capable of conception must be post-menopausal (one year or greater without menses), surgically incapable of childbearing, or practicing effective methods of birth control.

6. Male subjects must be practicing a medically accepted form of contraception during the study period. Abstinence is an acceptable method of contraception.

Exclusion Criteria

1. Subjects who have been enrolled in any kind of cell therapy within six months prior to screening visit (Visit 1).

2. Female subjects who have a positive pregnancy test result.

3. Subjects who have had severe vital organ diseases, including but not limited to cardiac (e.g., heart failure), liver (e.g., acute hepatic failure or chronic liver cirrhosis), lung (e.g., respiratory failure) and renal (e.g., hemodialysis or peritoneal dialysis) diseases, within six months prior to screening visit (Visit 1).

4. Subjects with immunological disorders (e.g., systemic lupus erythematosus), within six months prior to screening visit (Visit 1).

5. Subjects with other neurological disorders (e.g., Alzheimer's disease), within six months prior to screening visit (Visit 1).

6. Subjects who had received chemotherapy/radiotherapy within five years prior to screening visit (Visit 1).

7. Subjects with any history of malignant tumors.

8. Subjects with dementia or other psychiatric illnesses, including but not limited to disabling depression, bipolar disorder, schizophrenia.

9. Subjects with a Beck Depression Inventory Second Edition (BDI-II) score of over 20 points.

10. Subjects not suitable for this clinical trial according to investigator's judgment.

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

Sponsors

Steminent US, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Other Identifiers

IB02-US

Identifier Type: -

Identifier Source: org_study_id