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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03067831
• Other study ID #: SCA-DMD1
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: September 2015
• Est. completion date: December 2021

Study information

• Verified date: March 2020
• Source: Stem Cells Arabia
• Contact: Adeeb AlZoubi, Ph.D.
• Phone: 00962795337575
• Email: adeebalzoubi[@]stemcellsarabia.net
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: December 2021
• Est. primary completion date: November 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 25 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Biological:
• Stem Cells
Transplantation of purified autologous bone marrow-derived stem cells.

Locations

Country	Name	City	State
Jordan	Stem Cells of Arabia	Amman

Sponsors (1)

Lead Sponsor	Collaborator
Stem Cells Arabia

Country where clinical trial is conducted

Jordan, 

References & Publications (4)

Carletti B, Piemonte F, Rossi F. Neuroprotection: the emerging concept of restorative neural stem cell biology for the treatment of neurodegenerative diseases. Curr Neuropharmacol. 2011 Jun;9(2):313-7. doi: 10.2174/157015911795596603. — View Citation

Farini A, Villa C, Manescu A, Fiori F, Giuliani A, Razini P, Sitzia C, Del Fraro G, Belicchi M, Meregalli M, Rustichelli F, Torrente Y. Novel insight into stem cell trafficking in dystrophic muscles. Int J Nanomedicine. 2012;7:3059-67. doi: 10.2147/IJN.S30595. Epub 2012 Jun 20. — View Citation

Maclean S, Khan WS, Malik AA, Anand S, Snow M. The potential of stem cells in the treatment of skeletal muscle injury and disease. Stem Cells Int. 2012;2012:282348. doi: 10.1155/2012/282348. Epub 2011 Dec 19. — View Citation

Sienkiewicz D, Kulak W, Okurowska-Zawada B, Paszko-Patej G, Kawnik K. Duchenne muscular dystrophy: current cell therapies. Ther Adv Neurol Disord. 2015 Jul;8(4):166-77. doi: 10.1177/1756285615586123. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improvement in muscle strength using Kinetics Muscle testing or MMT		12 months
Secondary	Brooke and Vignos Scale		12 months