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

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by Chaitanya Hospital, Pune
Sponsor:
Information provided by (Responsible Party):
Dr. Sachin Jamadar, Chaitanya Hospital, Pune
ClinicalTrials.gov Identifier:
NCT01834040
First received: February 26, 2013
Last updated: September 16, 2014
Last verified: September 2014
  Purpose

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

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Chaitanya Hospital, Pune:

Primary Outcome Measures:
  • Improvement of daily living scale. [ Time Frame: 6 MONTH ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Improvement of Muscular dystrophy specific functional Rating scale [ Time Frame: 6 Months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 30
Study Start Date: September 2014
Estimated Study Completion Date: October 2016
Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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

  Eligibility

Ages Eligible for Study:   4 Years to 20 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party: Dr. Sachin Jamadar, CO-Investigator, Chaitanya Hospital, Pune
ClinicalTrials.gov Identifier: NCT01834040     History of Changes
Other Study ID Numbers: 00102
Study First Received: February 26, 2013
Last Updated: September 16, 2014
Health Authority: India: Institutional Review Board

Keywords provided by Chaitanya Hospital, Pune:
Duchenne Muscular Dystrophy
Muscular Dystrophy
Myopathy

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

ClinicalTrials.gov processed this record on October 22, 2014