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

Phase 1

• Conditions: Muscular Dystrophy; Duchenne Muscular Dystrophy,
• Interventions: Biological: Stem Cell

• Registration Number: NCT01834066
• Lead Sponsor: Chaitanya Hospital, Pune

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.

Study Timeline

• Study First Submitted: 2013-02-26
• Study First Submitted QC: 2013-04-15
• Study First Posted: 2013-04-17
• Status Verified: 2014-09
• Study Start: 2014-09
• Last Update Submitted: 2014-09-16
• Last Update Posted: 2014-09-17
• Primary Completion: 2016-11
• Study Completion: 2016-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 25

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
STEM CELL	Stem Cell	Intra thecal transplantation of autologous Stem Cell MNCs

Primary Outcome Measures

Name	Time	Method
Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score	6 Months

Secondary Outcome Measures

Name	Time	Method
-Improvement of daily living scale and baseline in EMG(electromyography)	6 months

Trial Locations

Locations (1)

Chaitnany Hospital; 🇮🇳 Pune, Maharashtra, India