Intramuscular Transplantation of Autologous Muscle Derived Stem Cell(MDSC) and Adipose Derived Mesenchymal Stem Cells (AD-MSC) in Patients With Facioscapulohumeral Dystrophy (FSHD), Phase I Clinical Trial
试验速览
- 阶段
- 1 期
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Myalsia
研究概览
简要总结
Facioscapulohumeral Dystrophy is a Autosomal dominant inherited dystrophy with the prevalence of 1:20,000 and it is the third most common dystrophy after the dystrophinopathies and myotonic dystrophy. The symptoms including: Pain, facial weakness, scapular fixator, humeral, truncal, pelvic girdle and lower-extremity weakness, High frequency hearing loss, Retinal telangiectasia . The existing treatments are not effective so, cell therapy is a new hope to improve patients' quality of life. Therefore, We design this clinical trial to evaluate the safety and feasibility of stem cell transplantation.
详细描述
In this study, we select 15 patients with FSHD based on eligibility criteria. All the patients underwent physical examination, laboratory evaluations, EMG-NCV, muscle sonography and muscle MRI. Then, a sample of patient's muscle is taken from Biceps Femoralis to isolate and culture of MDSC. The AD-MSC is prepared from Royan Adipose Tissue Bank. The patient is admitted in general hospital, and the cell suspensions are injected into biceps, triceps and trapezoids muscles by neurologists. After transplantation, the patients will be under observation for 5 hours and will be discharged if no side effect happen. All the patients will be followed at 1,2,4,6 and 12 months after cell injection.
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
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Both gender
- •Weakness of face muscle
- •FSHD phenotype positive
- •Genetic Test confirmation for FSHD
排除标准
- •Co - morbidities: Heart & respiratory diseases,malignancy, rheumatologic disorders
- •Progressive form of disease
- •Not able to sign the consent
结局指标
主要结局
Myalsia
时间窗: 1month
Evaluation the presence of myalsia 1month after cell injection.
Mass formation
时间窗: 6 months
Evaluation the probability of mass formation 6 months after cell injection.
次要结局
- CPK(1month)
- Muscle bulk(6 months)
- Hematoma(1 month)