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

September 16, 2014 updated by: Dr. Sachin Jamadar, Chaitanya Hospital, Pune

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

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,

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • India
    • Maharashtra
      • Pune, Maharashtra, India, 411009
        • Recruiting
        • Chaitanya Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years to 20 years (Child, Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: intralesional and Intravenous
Intralesional/ Intravenous of Autologous Stem cells.
Other: Intralesional/ Intravenous of Autologous Stem cells.
Intralesional/ Intravenous of Autologous MNCs per dose
Other Names:
  • Intralesional/ Intravenous of Autologous MNCs.
  • Intralesional/ Intravenous of Autologous MNCs

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Improvement of daily living scale.
Time Frame: 6 MONTH
6 MONTH

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chaitanya Hospital, Pune

Investigators

  • Principal Investigator: ANANT E BAGUL, M.S, Chaitanya Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2014

Primary Completion (Anticipated)

September 1, 2016

Study Completion (Anticipated)

October 1, 2016

Study Registration Dates

First Submitted

February 26, 2013

First Submitted That Met QC Criteria

April 15, 2013

First Posted (Estimate)

April 17, 2013

Study Record Updates

Last Update Posted (Estimate)

September 17, 2014

Last Update Submitted That Met QC Criteria

September 16, 2014

Last Verified

September 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 00102

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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