A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 45
- 主要终点
- Scale for the assessment anf rating of ataxia (SARA)
研究概览
简要总结
The purpose of this study is verify the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) therapy for patients with Spinocerebellar Ataxia, and in addition, explore the possible mechanisms of UC-MSC therapy in Spinocerebellar Ataxia.
详细描述
This is a random, open label, and parallel controled experiment. 45 patients are selected and sign consent forms, then divided into three groups. Doctors collect the basic information of patient (including age,height, mental condition, vital sign, history of disease, pharmaco-history, genotype test, and so on.) All patients receive laboratory and image examination as baseline. Then, they will give cell treatment based on the clinical protocol. Doctors have follow-up visit on 1, 2, 3, 6, and 12 months after treatment, and do efficacy evaluation.
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
- None
入排标准
- 年龄范围
- 16 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical and genomic test diagnoses as spinocerebellar ataxias (SCA) (include SCA 1, SCA 2, SCA 3, SCA 6), SARA (Scale for Assessment and Rating of Ataxia) score is 2-5, can complete 8-meter walking test（8MW）
- •Do not receive stem cells treatment in 6 months
- •Participants sign the consent form based on the experiment process and statement
排除标准
- •Cardiac, renal, hepatic insufficiency; total bilirubin is 1.5 times higher than normal value, aspartate transaminase（AST）or alanine aminotransferase（ALT）is 2.5 times higher than normal value
- •Hemogram: total white blood cells <3.0 * 10^9 cells/L, blood platelet <75 * 10^9/L, hemoglobin <100g/L
- •pneumonia, or severe infection
- •With severe allergic history
- •Brain organic disorder, like brain tumor
- •Serum with HIV, syphilis antibody positive
- •Severe mental disease, cognitive disorder patients
- •Other severe system or organ organic disease
- •Pregnant, breast feeding, or planning pregnant women
- •Participate other clinical experiments in 3 months
- •With some other conditions that doctor propose not to participate
研究组 & 干预措施
Control groups
No intervention
Intravenous infusion group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
干预措施: umbilical cord mesenchymal stem cell (Biological)
Intrathecal injection group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
干预措施: umbilical cord mesenchymal stem cell (Biological)
Intravenous infusion group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
干预措施: Intravenous infusion (Procedure)
Intrathecal injection group
Umbilical cord mesenchymal stem cells (SCLnow 19#)
干预措施: Intrathecal injection (Procedure)
结局指标
主要结局
Scale for the assessment anf rating of ataxia (SARA)
时间窗: 12 months
Based on SARA score to calculate treatment efficacy. The equation is as follow, Efficacy = (patients who accepted treatment are effective/patients who accepted treatment)\* 100% Effective: after 12 months, score of patient decrease 1 point or more; Ineffective: after 12 months, score decrease less than 1 point or increase.
次要结局
- Image examinations(12 months)
- Inventory of Non-Ataxia Symptoms (INAS) score(12 months)
- Cerebrospinal fluid (csf) routine(12 months)