Clinical trial NCT02285673: Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy
Official title: Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy: Phase 1/2 Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: umbilical cord, mesenchymal stem cell
Lead sponsor: Acibadem University
Sponsor class: OTHER
First posted: 2014-11-07
Enrollment: 10
Sex: MALE
Minimum age (years): 7
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Duchenne muscular dystrophy gene expression
timeFrame: up to 9 months
Secondary outcomes:
Other outcomes:
Locations:
city: Istanbul
country: Turkey
facility: Acibadem Labcell
state: Uskudar
Study officials:
References:
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