NCT02540655已完成2 期
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
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 56
- 试验地点
- 1
- 主要终点
- To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 20 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects are with genotypically confirmed spinocerebellar ataxia type 2 or spinocerebellar ataxia type
- •Subjects' baseline SARA score are in the range of 8 to
- •Subjects are between 20 and 70 years of age.
- •Subjects who had signed informed consent.
排除标准
- •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.
结局指标
主要结局
To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.
时间窗: 14 months
次要结局
- To assess the changes from the baseline to all visits on inventory of non-ataxia signs (INAS)(14 months)
- To assess the changes from the baseline to all visits on sensory organization test (SOT)(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).(14 months)
- To assess the changes from the baseline to all visits on SCA functional index (SCAFI)(14 months)
- To assess the changes from the baseline to all visits on clinical global impression (CGI)(14 months)
- To assess the changes from the baseline to all visits on patient global impression of improvement (PGI-I)(14 months)
研究者
研究点 (1)
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