NCT01834040

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

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

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Chaitanya Hospital, Pune · Academic / Other
• Sex: All
• Age: 4 Years – 20 Years
• Healthy volunteers: Accepted

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

Conditions

• Muscular Dystrophy
• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
OTHER	Intralesional/ Intravenous of Autologous Stem cells.	Intralesional/ Intravenous of Autologous MNCs per dose

Timeline

• Start date: 2014-09-01
• Primary completion: 2016-09-01
• Completion: 2016-10-01
• First posted: 2013-04-17
• Last updated: 2014-09-17

Locations

1 site across 1 country: India