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

Muscular Dystrophy Clinical Trial

— mdp  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01834066
• Other study ID #: 00101
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: February 26, 2013
• Last updated: September 16, 2014
• Start date: September 2014
• Est. completion date: December 2016

Study information

• Verified date: September 2014
• Source: Chaitanya Hospital, Pune
• Contact: Sachin P Jamadar, D.Ortho
• Phone: +918888788880
• Email: sac2751982[@]gmail.com
• Is FDA regulated: No
• Health authority: India: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. completion date: December 2016
• Est. primary completion date: November 2016
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 6 Years to 25 Years

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy,
• Muscular Dystrophies
• Muscular Dystrophy
• Muscular Dystrophy, Duchenne

Intervention

Biological:
• Stem Cell
Intralesional transfer of Autologous Stem cell (MNCs) per dose. 6 doses in 3 months

Locations

Country	Name	City	State
India	Chaitnany Hospital	Pune	Maharashtra

Sponsors (1)

Lead Sponsor	Collaborator
Chaitanya Hospital, Pune

Country where clinical trial is conducted

India, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score		6 Months	Yes
Secondary	-Improvement of daily living scale and baseline in EMG(electromyography)		6 months	Yes