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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03067831
Recruitment Status : Recruiting
First Posted : March 1, 2017
Last Update Posted : March 17, 2020
Information provided by (Responsible Party):
Stem Cells Arabia

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).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Biological: Stem Cells Phase 1 Phase 2

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).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Purified Autologous Bone Marrow-Derived Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy.
Study Start Date : September 2015
Estimated Primary Completion Date : November 2021
Estimated Study Completion Date : December 2021

Arm Intervention/treatment
Experimental: Stem Cells
Transplantation of purified autologous bone marrow-derived stem cells.
Biological: Stem Cells
Transplantation of purified autologous bone marrow-derived stem cells.

Primary Outcome Measures :
  1. Improvement in muscle strength using Kinetics Muscle testing or MMT [ Time Frame: 12 months ]

Secondary Outcome Measures :
  1. Brooke and Vignos Scale [ Time Frame: 12 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03067831

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Contact: Adeeb AlZoubi, Ph.D. 00962795337575

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Stem Cells of Arabia Recruiting
Amman, Jordan, 11953
Contact: Adeeb AlZoubi, PhD   
Principal Investigator: Adeeb AlZoubi, PhD         
Sponsors and Collaborators
Stem Cells Arabia
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Responsible Party: Stem Cells Arabia Identifier: NCT03067831    
Other Study ID Numbers: SCA-DMD1
First Posted: March 1, 2017    Key Record Dates
Last Update Posted: March 17, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Stem Cells Arabia:
stem cells
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked