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

Duchenne Muscular Dystrophy Clinical Trial

— 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01834040
• Other study ID #: 00102
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: February 26, 2013
• Last updated: September 16, 2014
• Start date: September 2014
• Est. completion date: October 2016

Study information

• Verified date: September 2014
• Source: Chaitanya Hospital, Pune
• Contact: Sachin S Jamadar, Dortho
• Phone: +918888788880
• Email: sac2751982[@]gmail.com
• Is FDA regulated: No
• Health authority: India: Institutional Review Board
• Study type: Interventional

Clinical Trial 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,

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: October 2016
• Est. primary completion date: September 2016
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 4 Years to 20 Years

Eligibility

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 Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy
• Muscular Dystrophy, Duchenne

Intervention

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

Locations

Country	Name	City	State
India	Chaitanya Hospital	Pune	Maharashtra

Sponsors (1)

Lead Sponsor	Collaborator
Chaitanya Hospital, Pune

Country where clinical trial is conducted

India, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improvement of daily living scale.		6 MONTH	Yes
Secondary	Improvement of Muscular dystrophy specific functional Rating scale		6 Months	Yes