Stemchymal® for Polyglutamine Spinocerebellar Ataxia

NCT06397274 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: IB02-US
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (1)

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

  6 months

Study Arms (2)

Stemchymal®

EXPERIMENTAL

Biological: Stemchymal

Placebo

PLACEBO COMPARATOR

Biological: Placebo

Interventions

Stemchymal BIOLOGICAL

Patients will receive Stemchymal® through intravenous infusion

Stemchymal®

Placebo BIOLOGICAL

Patients will receive Placebo through intravenous infusion

Placebo

Eligibility Criteria

• Age: 20 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects are with genotypically confirmed SCA3.
• Subjects' SARA scores are in the range of 5 to15.
• Subjects are between 20 and 70 years of age.
• Subjects who have signed informed consent.
• 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.
• Male subjects must be practicing a medically accepted form of contraception during the study period. Abstinence is an acceptable method of contraception.

You may not qualify if:

• Subjects who have been enrolled in any kind of cell therapy within six months prior to screening visit (Visit 1).
• Female subjects who have a positive pregnancy test result.
• 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).
• Subjects with immunological disorders (e.g., systemic lupus erythematosus), within six months prior to screening visit (Visit 1).
• Subjects with other neurological disorders (e.g., Alzheimer's disease), within six months prior to screening visit (Visit 1).
• Subjects who had received chemotherapy/radiotherapy within five years prior to screening visit (Visit 1).
• Subjects with any history of malignant tumors.
• Subjects with dementia or other psychiatric illnesses, including but not limited to disabling depression, bipolar disorder, schizophrenia.
• Subjects with a Beck Depression Inventory Second Edition (BDI-II) score of over 20 points.
• Subjects not suitable for this clinical trial according to investigator's judgment.

Sponsors & Collaborators

• Steminent US, Inc.: lead

MeSH Terms

Conditions

Spinocerebellar Ataxias

Condition Hierarchy (Ancestors)

Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 29, 2024
• First Posted: May 2, 2024
• Study Start: June 1, 2025
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2028
• Last Updated: May 2, 2024

Record last verified: 2024-04