Stemchymal® for Polyglutamine Spinocerebellar Ataxia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Stemchymal, Placebo.
- Who it may be relevant to
- Registry conditions: Spinocerebellar Ataxias. Basic parameters: 20 years — 70 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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
Overview
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.
Interventions
- Biological Stemchymal
Patients will receive Stemchymal® through intravenous infusion - Biological Placebo
Patients will receive Placebo through intravenous infusion
Primary outcome measures
- 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]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Triple blind
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT06397274 · IB02-US