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

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Disease Information

Descriptive Information
Brief Title † Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
Official Title † Safety and Efficacy of Purified Autologous Bone Marrow-Derived Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy.
Brief Summary 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 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).
Study Phase Phase 1/Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Improvement in muscle strength using Kinetics Muscle testing or MMT
Secondary Outcome Measure † Brooke and Vignos Scale
Condition † Duchenne Muscular Dystrophy
Intervention † BiologicalStem Cells
Study Arms / Comparison Groups Stem Cells Transplantation of purified autologous bone marrow-derived stem cells.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Biological
Estimated Enrollment † 20
Start Date † September 2015
Completion Date March 2020
Primary Completion Date December 2019
Eligibility Criteria † 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
Gender All
Ages 4 Years - 25 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries †
Administrative Information
NCT ID † NCT03067831
Organization ID SCA-DMD1
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Stem Cells Arabia
Collaborators ††
Investigators † : ,
Information Provided By
Verification Date October 2016
First Received Date † February 25, 2017
Last Updated Date March 1, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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