Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy

Phase 1

• Conditions: Duchenne Muscular Dystrophy

• Registration Number: NCT02285673
• Lead Sponsor: Acibadem University

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-11
• Status Verified: 2014-11
• Study First Submitted: 2014-11-05
• Study First Submitted QC: 2014-11-06
• Last Update Submitted: 2014-11-06
• Study First Posted: 2014-11-07
• Last Update Posted: 2014-11-07
• Primary Completion: 2015-02
• Study Completion: 2015-11

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: Male
• Target Recruitment: 10

Inclusion Criteria

• Patients with diagnosis of DMD that is proven clinically and genetically Age between 7-20 Patients need partial respiratory support, during the day Patients have less than or equal to stage I NIH, Liver, renal and cardiac function Patients without cancer Patients without allergic disease Patients without bleeding diathesis,

Exclusion Criteria

Patients need complete respiratory support Patients have more than to stage II NIH, Liver, renal and cardiac function Patients have bleeding diathesis and allergic disease

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Duchenne muscular dystrophy gene expression	up to 9 months

Trial Locations

Locations (1)

Acibadem Labcell; 🇹🇷 Istanbul, Uskudar, Turkey