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

Chaitanya Hospital, Pune

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Duchenne Muscular Dystrophy,; Muscular Dystrophy

Treatments: Biological: Stem Cell

Study type: Interventional

Funder types: Other

Identifiers

NCT01834066

00101

Details and patient eligibility

About

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.

Full 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.

Enrollment: 25 estimated patients

Sex: All

Ages: 6 to 25 years old

Volunteers: Accepts Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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25 participants in 1 patient group

STEM CELL

Other group

Description:

Intra thecal transplantation of autologous Stem Cell MNCs

Treatment:

Biological: Stem Cell