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Completed

NCT Number: NCT02540655

Efficacy and Safety Study of Stemchymal® in 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 spinocerebellar ataxia type 2 or spinocerebellar ataxia type 3.
  • Subjects' baseline SARA score are in the range of 8 to15.
  • Subjects are between 20 and 70 years of age.
  • Subjects who had signed informed consent.

Exclusion criteria

  • Subjects had been enrolled in any kind of cell therapy within six months prior to screening visit.
  • Females with a positive pregnancy test result.
  • Subjects who had had severe vital organ disease as diagnosed, including but not limited to cardiac (ex. heart failure), liver (ex. acute hepatic failure or chronic liver cirrhosis), lung (ex. respiratory failure) and renal (ex. hemodialysis or peritoneal dialysis) insufficiency, within six months prior to screening visit.
  • Subjects with immunological disorders (ex. Systemic lupus erythematosus), within six months prior to screening visit.
  • Subjects with other neurological disorders (ex. Alzheimer's disease), within six months prior to screening visit.
  • Subjects who had received chemotherapy/radiotherapy within five years prior to screening visit.
  • Subjects with any history of malignancy tumors.
  • Subjects with a history of hypersensitivity/allergy to penicillin.
  • Subjects with dementia or other psychiatric illnesses, including but not limited to disabling depression, bipolar disorder, and schizophrenia.
  • Subjects with Beck Depression Inventory Second Edition (BDI-II) score over 20 points.
  • Subjects with unstable illnesses or contraindication for this clinical trial according to PI's judgment.

Treatment and study plan

Stemchymal®

Biological

Patients will receive Stemchymal® through intravenous infusion

Excipients

Procedure

Patients will receive excipients through intravenous infusion

Primary outcomes

  1. To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.

    Time frame: 14 months

Secondary outcomes

  1. To assess the safety by incidence of adverse events, changes of vital signs, blood biochemistry tests, complete blood count, immunoactivity assay, urinalysis and magnetic resonance imaging (MRI).

    Time frame: 14 months

  2. To assess the changes from the baseline to all visits on SCA functional index (SCAFI)

    Time frame: 14 months

  3. To assess the changes from the baseline to all visits on sensory organization test (SOT)

    Time frame: 14 months

    Balance test

  4. To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)

    Time frame: 14 months

  5. To assess the changes from the baseline to all visits on clinical global impression (CGI)

    Time frame: 14 months

  6. To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)

    Time frame: 14 months

Sponsors and collaborators

Lead sponsor

Steminent Biotherapeutics 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® Infusion for the Treatment of Polyglutamine Spinocerebellar Ataxia

Important dates

Study start
2015
Primary completion
2021
Study completion
2021
First posted
Sep 4, 2015
Registry last updated
Oct 27, 2022

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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