Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Activities of Daily Living(ADL)scale
研究概览
简要总结
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle. To a large extent, patients always lose motor function gradually and die for heart failure or severe infection at the end stage of DMD. At present, the treatment strategy relies on heteropathy accompanied with rehabilitation training. However, the therapeutic effect remains extremely limited.
Human umbilical cord mesenchymal stem cells (hUC-MSCs) have been evidenced to improve motor function, increase muscle strength and reduce abnormal levels of related enzymes, such as creatine kinase (CK), lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and aspartate aminotransferase (AST). This study is aimed to explore the safety and efficacy of hUC-MSCs transplantation for DMD.
详细描述
This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive muscular dystrophy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 5-12 years
- •Clinical manifestation, enzymology, electromyogram, gene type confirmed the diagnose of Duchenne muscular dystrophy
- •Sign the consent form and follow the clinic trail procedure
排除标准
- •Not Duchenne muscular dystrophy
- •Any history of hypersensitivity to serum products,or other know drug and food allergy
- •Combined Pneumonia or other Severe systemic bacteria infection
- •HIV+, TPPA +， patients diagnosed as HBV or HCV
- •Tumor Markers +
- •Severe psychotic patients, cognitive dysfunction
- •Coagulation disorders
- •Uncontrolled hypertension after treatment，blood pressure≥180mmHg/110 mmHg
- •Other severe systemic or organic disease
- •Enrollment in other trials in the last 3 months
- •Received any stem cell therapy in past 6 months
- •Other criteria that investigator consider improper for inclusion
结局指标
主要结局
Activities of Daily Living(ADL)scale
时间窗: 1 year after treatment
次要结局
- Incidences of Adverse Event and Serious Adverse Event(1 year after treatment)
- Change from baseline in CK(1 year after treatment)
- Change from baseline in LDH(1 year after treatment)
- Change from baseline in ALT(1 year after treatment)
- Change from baseline in AST(1 year after treatment)
- Change from baseline to manual muscle test(MMT)(1 year after treatment)
- Change from baseline in electromyography(EMG)(1 year after treatment)