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

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ClinicalTrials.gov Identifier: NCT01834040
Recruitment Status : Unknown
Verified September 2014 by Dr. Sachin Jamadar, Chaitanya Hospital, Pune.
Recruitment status was:  Recruiting
First Posted : April 17, 2013
Last Update Posted : September 17, 2014
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
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,

Condition or disease Intervention/treatment Phase
Muscular Dystrophy Duchenne Muscular Dystrophy Other: Intralesional/ Intravenous of Autologous Stem cells. Phase 1 Phase 2

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 Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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 Start Date : September 2014
Estimated Primary Completion Date : September 2016
Estimated Study Completion Date : October 2016


Arms and Interventions

Arm Intervention/treatment
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


Outcome Measures

Primary Outcome Measures :
  1. Improvement of daily living scale. [ Time Frame: 6 MONTH ]

Secondary Outcome Measures :
  1. Improvement of Muscular dystrophy specific functional Rating scale [ Time Frame: 6 Months ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01834040


Contacts
Contact: Sachin S Jamadar, Dortho +918888788880 sac2751982@gmail.com

Locations
India
Chaitanya Hospital Recruiting
Pune, Maharashtra, India, 411009
Contact: Sachin S Jamadar, D ORTHO    +918888788880    sac2751982@gmail.com   
Sponsors and Collaborators
Chaitanya Hospital, Pune
Investigators
Principal Investigator: ANANT E BAGUL, M.S CHAITANYA HOSPITAL
More Information

Responsible Party: Dr. Sachin Jamadar, CO-Investigator, Chaitanya Hospital, Pune
ClinicalTrials.gov Identifier: NCT01834040     History of Changes
Other Study ID Numbers: 00102
First Posted: April 17, 2013    Key Record Dates
Last Update Posted: September 17, 2014
Last Verified: September 2014

Keywords provided by Dr. Sachin Jamadar, Chaitanya Hospital, Pune:
Duchenne Muscular Dystrophy
Muscular Dystrophy
Myopathy

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked