Study Safety and Efficacy of of AUTOLOGOUS Bone Marrow Derived Mono Nuclear Stem Cell (BMMNCs) for the Patient With Duchenne 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
- 30
- 试验地点
- 2
- 主要终点
- Improvement of daily living scale.
研究概览
简要总结
This Study is single arm, single center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy,
详细描述
Muscular dystrophies, or MD, are a group of inherited conditions, which means they are passed down through families. They may occur in childhood or adulthood. There are many different types of muscular dystrophy. They include:
Duchenne muscular dystrophy is a form of muscular dystrophy that worsens quickly. Other muscular dystrophy (including Becker's muscular dystrophy) get worse much more slowly.
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.
Symptoms usually appear before age 6 and may appear as early as infancy. They may include:
Fatigue
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
- 4 Years 至 20 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 4 to 20 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 with History of Immunodeficiency HIV+,Hepatitis B ,HBV and TPPA+, Tumor Markers+
- •Patients with History of Hypertension and Hypersensitive.
- •Patient who is not Diagnose of Duchenne Muscular Dystrophy.
- •Alcohol and drug abuse / dependence.
结局指标
主要结局
Improvement of daily living scale.
时间窗: 6 MONTH
次要结局
- Improvement of Muscular dystrophy specific functional Rating scale(6 Months)
研究者
Dr. Sachin Jamadar
CO-Investigator
Chaitanya Hospital, Pune