Clinical trial NCT03067831: 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.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: stem cells
Lead sponsor: Stem Cells Arabia
Sponsor class: OTHER
First posted: 2017-03-01
Enrollment: 20
Sex: ALL
Minimum age (years): 4
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

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 \< 30%
* Pregnancy or breastfeeding

Primary outcomes:
measure: Improvement in muscle strength using Kinetics Muscle testing or MMT
timeFrame: 12 months
Secondary outcomes:
measure: Brooke and Vignos Scale
timeFrame: 12 months
Other outcomes:
Locations:
city: Amman
country: Jordan
facility: Stem Cells of Arabia
Study officials:
References:
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.
citation: 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.
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.
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.