Stemchymal® for Polyglutamine Spinocerebellar Ataxia

A Phase II, Randomized, Double-Blind, Placebo-controlled, Single-Center Study to Evaluate the Safety and Efficacy of Stemchymal (Allogeneic Adipose-derived Mesenchymal Stem Cells) Infusion for the Treatment of PolyQ Spinocerebellar Ataxia

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Spinocerebellar Ataxias
• Intervention / Treatment: Biological: Stemchymal; Biological: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Biological: Placebo; Patients will receive Placebo through intravenous infusion
Experimental: Stemchymal®	Biological: Stemchymal; Patients will receive Stemchymal® through intravenous infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change of Scale for the Assessment and Rating of Ataxia (SARA) scores from baseline (week 0) at 6 months (week 24).	6 months

Collaborators and Investigators

• Sponsor: Steminent US, Inc.

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2025
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2028

Study Registration Dates

• First Submitted: April 29, 2024
• First Submitted That Met QC Criteria: April 29, 2024
• First Posted (Estimated): May 2, 2024

Study Record Updates

• Last Update Posted (Estimated): May 2, 2024
• Last Update Submitted That Met QC Criteria: April 29, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations
• Other Study ID Numbers: IB02-US

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No