Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy.

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Safety and Efficacy of Bone Marrow Autologous Cells in Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial

Conditions
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Muscular Dystrophy, Duchenne Muscular Dystrophy,

Intervention / Treatment
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* Biological: Stem Cell


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: STEM CELL<br>Intra thecal transplantation of autologous Stem Cell MNCs | Biological: Stem Cell<br>* Intralesional transfer of Autologous Stem cell (MNCs) per dose. 6 doses in 3 months<br>* Other names: Intravenous transfer of Autologous Stem Cell ( MNCs );|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score |  | 6 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| -Improvement of daily living scale and baseline in EMG(electromyography) |  | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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muscular Dystrophy stem cell