Safety and Efficacy of Bone Marrow Autologous Cells in Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 25
- 试验地点
- 2
- 主要终点
- Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score
研究概览
简要总结
This Study is single arm, single centre trial to check the safety and efficacy of Bone Marrow derived autologous cell(100 million per dose) for the patient with Duchenne Muscular Dystrophy.
详细描述
Muscular dystrophy is a group of inherited disorders that involve muscle weakness and loss of muscle tissue, which get worse over time. Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the muscles). However, it often occurs in people without a known family history of the condition. slowly progress diseases.it causes Muscle weaknesses, Difficulty with motor skills ,Progressive difficulty walking.Breathing difficulties and heart disease,Frequent falls,weak limbs,lose motor Function.Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other areas of the body.Trouble getting up from a lying position or climbing stairs.
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
- 6 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patient with Diagnose of Duchenne Muscular Dystrophy.
- •Aged in between 6 to 25 Years.
- •Willingness to undergo Bone Marrow derived Autologous cell Therapy.
- •Ability to comprehend the explained protocol and thereafter give an informed consent as well as sign the required Informed Consent form(ICF) for the study.
- •Ability and willingness to regular visit to hospital for protocol procedures and follow up
排除标准
- •Patient who is not Diagnose of Duchenne Muscular Dystrophy.
- •Patient with History of Immunodeficiency HIV+,Hepatitis B ,HBV and TPPA+,Tumor Markers+
- •History of Life threatening allergic or immune -Mediated Reaction.
- •the site of bone marrow aspiration potentially limiting Procedure.
- •Alcohol and drug abuse / dependence.
- •Patients with History of Hypertension and Hypersensitive.
结局指标
主要结局
Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score
时间窗: 6 Months
次要结局
- -Improvement of daily living scale and baseline in EMG(electromyography)(6 months)
研究者
Dr. Sachin Jamadar
C0- Investigator
Chaitanya Hospital, Pune