Stemchymal® for Polyglutamine Spinocerebellar Ataxia

Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age20-70
SponsorSteminent US, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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).

Trial design

Design model

Parallel

Treatments tested in this trial

  • Stemchymal

    Biological/Vaccine

    Patients will receive Stemchymal® through intravenous infusion

  • Placebo

    Biological/Vaccine

    Patients will receive Placebo through intravenous infusion

Treatment groups

20 Participants
are divided into 2 treatment groups
Group A: Stemchymal®Experimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

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

Secondary outcomes

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

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