Duchenne Muscular Dystrophy,, Muscular Dystrophy
Conditions
Keywords
muscular Dystrophy stem cell
Brief summary
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
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.
Interventions
BIOLOGICALStem Cell
Intralesional transfer of Autologous Stem cell (MNCs) per dose. 6 doses in 3 months
Sponsors
Chaitanya Hospital, Pune
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 25 Years
Healthy volunteers
Yes
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score | 6 Months |
Secondary
| Measure | Time frame |
|---|---|
| -Improvement of daily living scale and baseline in EMG(electromyography) | 6 months |
Countries
India
Contacts
Primary ContactSachin P Jamadar, D.Ortho
Outcome results
None listed