Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy

Overview

This Study is single arm, single center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy,

Full Title of Study: “Study Safety and Efficacy of of AUTOLOGOUS Bone Marrow Derived Mono Nuclear Stem Cell (BMMNCs) for the Patient With Duchenne Muscular Dystrophy. It is Self Funded (Patients’ Own Funding) Clinical Trial”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: September 2016

Detailed Description

Muscular dystrophies, or MD, are a group of inherited conditions, which means they are passed down through families. They may occur in childhood or adulthood. There are many different types of muscular dystrophy. They include: Duchenne muscular dystrophy is a form of muscular dystrophy that worsens quickly. Other muscular dystrophy (including Becker's muscular dystrophy) get worse much more slowly. 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. Symptoms usually appear before age 6 and may appear as early as infancy. They may include: Fatigue Learning difficulties (the IQ (intelligence quotient )can be below 75) Intellectual disability (possible, but does not get worse over time) Muscle weakness Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other areas of the body Difficulty with motor skills (running, hopping, jumping) Frequent falls Trouble getting up from a lying position or climbing stairs Weakness quickly gets worse Progressive difficulty walking Ability to walk may be lost by age 12, and the child will have to use a wheelchair Breathing difficulties and heart disease usually start by age 20

Interventions

  • Other: Intralesional/ Intravenous of Autologous Stem cells.
    • Intralesional/ Intravenous of Autologous MNCs per dose

Arms, Groups and Cohorts

  • Other: intralesional and Intravenous
    • Intralesional/ Intravenous of Autologous Stem cells.

Clinical Trial Outcome Measures

Primary Measures

  • Improvement of daily living scale.
    • Time Frame: 6 MONTH

Secondary Measures

  • Improvement of Muscular dystrophy specific functional Rating scale
    • Time Frame: 6 Months

Participating in This Clinical Trial

Inclusion Criteria

  • Patient with Diagnose of Duchenne Muscular Dystrophy. – Aged in between 4 to 20 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 with History of Immunodeficiency HIV+,Hepatitis B ,HBV and TPPA+, Tumor Markers+ – Patients with History of Hypertension and Hypersensitive. – Patient who is not Diagnose of Duchenne Muscular Dystrophy. – Alcohol and drug abuse / dependence.

Gender Eligibility: All

Minimum Age: 4 Years

Maximum Age: 20 Years

Are Healthy Volunteers Accepted: Accepts Healthy Volunteers

Investigator Details

  • Lead Sponsor
    • Chaitanya Hospital, Pune
  • Provider of Information About this Clinical Study
    • Principal Investigator: Dr. Sachin Jamadar, CO-Investigator – Chaitanya Hospital, Pune
  • Overall Official(s)
    • ANANT E BAGUL, M.S, Principal Investigator, CHAITANYA HOSPITAL
  • Overall Contact(s)
    • Sachin S Jamadar, Dortho, +918888788880, sac2751982@gmail.com

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