Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy

Acibadem University

Status and phase

Unknown

Phase 2

Phase 1

Conditions

Duchenne Muscular Dystrophy

Treatments

Biological: Umbilical Cord Mesenchymal Stem Cell

Study type

Interventional

Funder types

Other

Identifiers

NCT02285673

DMD-UC-MSC-1

Details and patient eligibility

About

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.

Enrollment

10 estimated patients

Sex

Male

Ages

7 to 20 years old

Volunteers

No Healthy Volunteers

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