Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
Safety and Efficacy of Purified Autologous Bone Marrow-Derived Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy.
1 other identifier
interventional
20
1 country
1
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Sep 2015
Longer than P75 for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2015
CompletedFirst Submitted
Initial submission to the registry
February 25, 2017
CompletedFirst Posted
Study publicly available on registry
March 1, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2021
CompletedMarch 17, 2020
March 1, 2020
6.2 years
February 25, 2017
March 15, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Improvement in muscle strength using Kinetics Muscle testing or MMT
12 months
Secondary Outcomes (1)
Brooke and Vignos Scale
12 months
Study Arms (1)
Stem Cells
EXPERIMENTALTransplantation of purified autologous bone marrow-derived stem cells.
Interventions
Transplantation of purified autologous bone marrow-derived stem cells.
Eligibility Criteria
You may qualify if:
- Age group of 3-25 years
- Duchenne muscular dystrophy diagnosed on the basis of clinical presentation
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Stem Cells of Arabia
Amman, 11953, Jordan
Related Publications (4)
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.
PMID: 26136844BACKGROUNDCarletti 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.
PMID: 22131940BACKGROUNDFarini 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.
PMID: 22787400BACKGROUNDMaclean 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.
PMID: 22220178BACKGROUND
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 25, 2017
First Posted
March 1, 2017
Study Start
September 1, 2015
Primary Completion
November 1, 2021
Study Completion
December 1, 2021
Last Updated
March 17, 2020
Record last verified: 2020-03