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

Phase 1

• Conditions: Duchenne Muscular Dystrophy; Muscular Dystrophy
• Interventions: Other: Intralesional/ Intravenous of Autologous Stem cells.

• Registration Number: NCT01834040
• Lead Sponsor: Chaitanya Hospital, Pune

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

Study Timeline

• Study First Submitted: 2013-02-26
• Study First Submitted QC: 2013-04-15
• Study First Posted: 2013-04-17
• Status Verified: 2014-09
• Study Start: 2014-09
• Last Update Submitted: 2014-09-16
• Last Update Posted: 2014-09-17
• Primary Completion: 2016-09
• Study Completion: 2016-10

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
intralesional and Intravenous	Intralesional/ Intravenous of Autologous Stem cells.	Intralesional/ Intravenous of Autologous Stem cells.

Primary Outcome Measures

Name	Time	Method
Improvement of daily living scale.	6 MONTH

Secondary Outcome Measures

Name	Time	Method
Improvement of Muscular dystrophy specific functional Rating scale	6 Months

Trial Locations

Locations (1)

Chaitanya Hospital; 🇮🇳 Pune, Maharashtra, India