Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy (BMMNC)
Primary Purpose
Muscular Dystrophy, Duchenne Muscular Dystrophy
Status
Unknown status
Phase
Phase 1
Locations
India
Study Type
Interventional
Intervention
Intralesional/ Intravenous of Autologous Stem cells.
Sponsored by
About this trial
This is an interventional treatment trial for Muscular Dystrophy focused on measuring Duchenne Muscular Dystrophy, Muscular Dystrophy, Myopathy
Eligibility 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.
Sites / Locations
- Chaitanya HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
intralesional and Intravenous
Arm Description
Intralesional/ Intravenous of Autologous Stem cells.
Outcomes
Primary Outcome Measures
Improvement of daily living scale.
Secondary Outcome Measures
Improvement of Muscular dystrophy specific functional Rating scale
Full Information
NCT ID
NCT01834040
First Posted
February 26, 2013
Last Updated
September 16, 2014
Sponsor
Chaitanya Hospital, Pune
1. Study Identification
Unique Protocol Identification Number
NCT01834040
Brief Title
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
Acronym
BMMNC
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 Type
Interventional
2. Study Status
Record Verification Date
September 2014
Overall Recruitment Status
Unknown status
Study Start Date
September 2014 (undefined)
Primary Completion Date
September 2016 (Anticipated)
Study Completion Date
October 2016 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Chaitanya Hospital, Pune
4. Oversight
Data Monitoring Committee
Yes
5. 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,
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy, Duchenne Muscular Dystrophy
Keywords
Duchenne Muscular Dystrophy, Muscular Dystrophy, Myopathy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
intralesional and Intravenous
Arm Type
Other
Arm Description
Intralesional/ Intravenous of Autologous Stem cells.
Intervention Type
Other
Intervention Name(s)
Intralesional/ Intravenous of Autologous Stem cells.
Other Intervention Name(s)
Intralesional/ Intravenous of Autologous MNCs., Intralesional/ Intravenous of Autologous MNCs
Intervention Description
Intralesional/ Intravenous of Autologous MNCs per dose
Primary Outcome Measure Information:
Title
Improvement of daily living scale.
Time Frame
6 MONTH
Secondary Outcome Measure Information:
Title
Improvement of Muscular dystrophy specific functional Rating scale
Time Frame
6 Months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sachin S Jamadar, Dortho
Phone
+918888788880
Email
sac2751982@gmail.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
ANANT E BAGUL, M.S
Organizational Affiliation
CHAITANYA HOSPITAL
Official's Role
Principal Investigator
Facility Information:
Facility Name
Chaitanya Hospital
City
Pune
State/Province
Maharashtra
ZIP/Postal Code
411009
Country
India
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sachin S Jamadar, D ORTHO
Phone
+918888788880
Email
sac2751982@gmail.com
12. IPD Sharing Statement
Learn more about this trial
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
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