Duchenne Muscular Dystrophy, Muscular Dystrophy
Conditions
Keywords
Duchenne Muscular Dystrophy, Muscular Dystrophy, Myopathy
Brief summary
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,
Detailed description
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 Learning difficulties (the IQ (intelligence quotient )can be below 75) Intellectual disability (possible, but does not get worse over time) Muscle weakness Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other areas of the body Difficulty with motor skills (running, hopping, jumping) Frequent falls Trouble getting up from a lying position or climbing stairs Weakness quickly gets worse Progressive difficulty walking Ability to walk may be lost by age 12, and the child will have to use a wheelchair Breathing difficulties and heart disease usually start by age 20
Interventions
Intralesional/ Intravenous of Autologous MNCs per dose
Sponsors
Study design
Eligibility
Inclusion criteria
* 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
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Improvement of daily living scale. | 6 MONTH |
Secondary
| Measure | Time frame |
|---|---|
| Improvement of Muscular dystrophy specific functional Rating scale | 6 Months |
Countries
India