Clinical Trials Logo
  1. Home
  2. Duchenne Muscular Dystrophy
  3. NCT03067831

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 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).

Clinical Trial 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

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03067831
Study type Interventional
Source Stem Cells Arabia
Contact Adeeb AlZoubi, Ph.D.
Phone 00962795337575
Email adeebalzoubi@stemcellsarabia.net
Status Recruiting
Phase Phase 1/Phase 2
Start date September 2015
Completion date December 2021
View Details
See also
  Status Clinical Trial Phase
Completed NCT05575648 - Dual Task in Duchenne Muscular Dystrophy N/A
Terminated NCT03907072 - Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy Phase 2/Phase 3
Not yet recruiting NCT06450639 - An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy Phase 2
Completed NCT04335942 - Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor N/A
Active, not recruiting NCT04906460 - Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) Phase 1/Phase 2
Active, not recruiting NCT02500381 - Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) Phase 3
Enrolling by invitation NCT05967351 - A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study Phase 3
Recruiting NCT01834040 - Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy Phase 1/Phase 2
Completed NCT02246478 - A Study of TAS-205 for Duchenne Muscular Dystrophy Phase 1
Active, not recruiting NCT01772043 - Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping N/A
Terminated NCT01168908 - Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Phase 2
Completed NCT00758225 - Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) Phase 2
Completed NCT03680365 - Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
Recruiting NCT03513367 - The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
Recruiting NCT05712447 - Duchenne Muscular Dystrophy Video Assessment Registry
Recruiting NCT01484678 - Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Completed NCT03319030 - Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
Terminated NCT01753804 - A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. N/A
Completed NCT02530905 - Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients Phase 1
Terminated NCT04708314 - An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy Phase 4