NCT02285673

Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy

Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy: Phase 1/2 Study

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Acibadem University · Academic / Other
• Sex: Male
• Age: 7 Years – 20 Years
• Healthy volunteers: Not accepted

Summary

Duchenne muscular dystrophy (DMD) is a genetic disorder caused by an absence of dystrophin and characterized by progressive muscle degeneration. There is no cure for DMD at present but, there are several strategies under-researched for treatment of DMD such as steroid treatment, gene theraphy, exon skipping, stop codon read through and gene repair, cell theraphy and theraphy with drug that help to produce utrophin protein. The aim of this study is investigate the eficacy of human umblical cord mesenchymal stem cells on DMD and understanding if wild type gene can be transfered to the patient.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
BIOLOGICAL	Umbilical Cord Mesenchymal Stem Cell

Timeline

• Start date: 2013-11-01
• Primary completion: 2015-02-01
• Completion: 2015-11-01
• First posted: 2014-11-07
• Last updated: 2014-11-07

Locations

1 site across 1 country: Turkey (Türkiye)