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NCT Number: NCT06397274

Stemchymal® for Polyglutamine 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.

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Key information

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Stemchymal

Biological

Patients will receive Stemchymal® through intravenous infusion

Placebo

Biological

Patients will receive Placebo through intravenous infusion

Primary outcomes

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

    Time frame: 6 months

Sponsors and collaborators

Lead sponsor

Steminent US, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
May 2, 2024
Registry last updated
May 2, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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