Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan
研究概览
简要总结
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements. Current treatments for Hereditary Ataxias are mainly pharmacological, rehabilitative, or psychological treatments，while no effective treatment available. Stem Cell therapy is a novel and promising therapeutic strategy for Hereditary Ataxias treatment. In this study, the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation will be evaluated in patients with Hereditary Ataxias.
详细描述
This Study is designed to evaluate the the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation in patients with Hereditary Ataxias.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 16-65 years.
- •Harding Diagnosis of SCAs, gene type confirmed.
- •Candidates who did not receive any stem cell therapy in past 6months.
- •sign the consent form and follow the clinic trail procedure.
排除标准
- •Cardiac insufficiency; Renal insufficiency; hepatic insufficiency; Total bilirubin higher than 1.5 times of upper limit of normal value; AST /ALT higher than 2.5 times of upper limit of normal value；
- •Routine Blood Test: WBC count <3.0×109/ L; PLT count <5×109/L ; or Hemoglobin <100g/L；
- •Combined Pneumonia or other Severe systemic bacteria infection；
- •Severe drug allergic history or anaphylaxis to 2 or more food or medicine；
- •Other brain organic disease (eg. Brain cancer)；
- •HIV+, Tumor Markers + ；
- •Severe psychotic patients, cognitive dysfunction， or can not understand or sign the Consent Form；
- •Other severe systemic or organic disease；
- •Uncontrolled hypertension，blood pressure≥180mmHg/110 mmHg after treatment；
- •Pregnancy；
- •Enrollment in other trials in the last 3 months；
- •Other criteria the investigator consider improper for inclusion.
结局指标
主要结局
the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan
时间窗: 1 year after treatment
scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale
时间窗: 1 year after treatment
次要结局
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability(1 year after treatment)
- Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability(1 year after treatment)