Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy

Study Overview
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Brief Summary
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This study is single arm, single center trial to study the safety and efficacy of bone marrow-derived autologous specific populations of stem cells and mesenchymal stem cells for the treatment of Duchenne Muscular Dystrophy (DMD).

Detailed Description
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Duchenne muscular dystrophy (DMD) is a genetically determined X-linked disease. The manifestation of muscle weakness typically starts around the age of 4-5 in males and deteriorates fast. Typically muscle loss occurs first in the upper legs and pelvis followed by muscles of the upper arms. It is caused by a mutation in the gene for the protein dystrophin. Dystrophin is crucial to maintain the muscle fiber cell membrane. Currently, there is no cure for muscular dystrophy. Corrective surgery, braces, and physical therapy may help with some of the symptoms. Assisted ventilation might be required in patients with weakness of breathing muscles. Medications prescribed include steroids to slow muscle degeneration, anti-convulsants to control seizures and muscle activity, and immunosuppressants to delay damage to muscle cells. For decades, research has been conducted to find an effective therapy for Duchenne muscular dystrophy (DMD). Stem cell based therapy is considered to be one of the most promising methods for treating muscular dystrophies. Stem cell based therapies for the treatment of Duchenne muscular dystrophy (DMD) can proceed via two strategies. The first is autologous stem cell transfer involving cells from a patient with Duchenne muscular dystrophy (DMD) that are genetically altered in vitro to restore dystrophin expression and are subsequently re-implanted. The second is allogenic stem cell transfer, containing cells from an individual with functional dystrophin, which are transplanted into a dystrophic patient. Herein, the investigators describe a method for the treatment of Duchenne muscular dystrophy (DMD) using autologous bone marrow derived specific populations of stem cells and mesenchymal stem cells transplanted in patients with Duchenne muscular dystrophy (DMD).

Official Title
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Safety and Efficacy of Purified Autologous Bone Marrow-Derived Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy.

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Biological: Stem Cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age group of 3-25 years Duchenne muscular dystrophy diagnosed on the basis of clinical presentation Exclusion Criteria: Respiratory Distress Acute infections such as Human Immunodeficient Virus/Hepatitis B Virus/Hepatitis C Virus malignancies Acute medical conditions such as respiratory infections, fever, hemoglobin less than 8 bleeding tendency, bone marrow disorder, left ventricular ejection fraction < 30% Pregnancy or breastfeeding

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Stem Cells<br>Transplantation of purified autologous bone marrow-derived stem cells. | Biological: Stem Cells<br>* Transplantation of purified autologous bone marrow-derived stem cells.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Improvement in muscle strength using Kinetics Muscle testing or MMT |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Brooke and Vignos Scale |  | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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stem cells