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

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ClinicalTrials.gov Identifier: NCT03067831
Recruitment Status : Active, not recruiting
First Posted : March 1, 2017
Last Update Posted : March 3, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
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).


Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
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 : December 2019
Estimated Study Completion Date : March 2020


Arms and Interventions

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.


Outcome Measures

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 ]

Eligibility Criteria

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
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 < 30%
  • Pregnancy or breastfeeding
More Information

Publications:
Responsible Party: Stem Cells Arabia
ClinicalTrials.gov Identifier: NCT03067831     History of Changes
Other Study ID Numbers: SCA-DMD1
First Posted: March 1, 2017    Key Record Dates
Last Update Posted: March 3, 2017
Last Verified: October 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Stem Cells Arabia:
stem cells

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked