Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia
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|ClinicalTrials.gov Identifier: NCT02540655|
Recruitment Status : Completed
First Posted : September 4, 2015
Last Update Posted : October 27, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Cerebellar Ataxia||Biological: Stemchymal® Procedure: Excipients||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||56 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official 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|
|Study Start Date :||September 2015|
|Actual Primary Completion Date :||February 26, 2021|
|Actual Study Completion Date :||February 26, 2021|
Infusion of Stemchymal®
Patients will receive Stemchymal® through intravenous infusion
Placebo Comparator: Vehicle
Infusion of excipients
Patients will receive excipients through intravenous infusion
- 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 ]
- 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 ]
- To assess the changes from the baseline to all visits on SCA functional index (SCAFI) [ Time Frame: 14 months ]
- To assess the changes from the baseline to all visits on sensory organization test (SOT) [ Time Frame: 14 months ]Balance test
- To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS) [ Time Frame: 14 months ]
- To assess the changes from the baseline to all visits on clinical global impression (CGI) [ Time Frame: 14 months ]
- To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I) [ Time Frame: 14 months ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||20 Years to 70 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02540655
|Taipei Veterans General Hospital|
|Responsible Party:||Steminent Biotherapeutics Inc.|
|Other Study ID Numbers:||
|First Posted:||September 4, 2015 Key Record Dates|
|Last Update Posted:||October 27, 2022|
|Last Verified:||October 2022|
Adult Stem Cells
Nervous System Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn