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

March 15, 2020 updated by: Stem Cells Arabia

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

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).

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

20

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Brooke and Vignos Scale
Time Frame: 12 months
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Stem Cells Arabia

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2015

Primary Completion (Anticipated)

November 1, 2021

Study Completion (Anticipated)

December 1, 2021

Study Registration Dates

First Submitted

February 25, 2017

First Submitted That Met QC Criteria

February 28, 2017

First Posted (Actual)

March 1, 2017

Study Record Updates

Last Update Posted (Actual)

March 17, 2020

Last Update Submitted That Met QC Criteria

March 15, 2020

Last Verified

March 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SCA-DMD1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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