临床试验/NCT02285673

NCT02285673Unknown1 期

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

Acibadem University2 个研究点分布在 1 个国家目标入组 10 人开始时间: 2013年11月1日最近更新: 2014年11月7日

适应症

试验速览

阶段: 1 期
发起方
入组人数: 10
试验地点: 2
主要终点: Duchenne muscular dystrophy gene expression

研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

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.

研究设计

研究类型: Interventional
分配方式: Randomized
干预模型: Single Group
主要目的: Treatment
盲法: None

入排标准

年龄范围: 7 Years 至 20 Years（Child, Adult）
性别: Male
接受健康志愿者: 否

入选标准

•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,

排除标准

•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

结局指标

主要结局

Duchenne muscular dystrophy gene expression

时间窗: up to 9 months

次要结局

未报告次要终点

研究者

发起方

Acibadem University

申办方类型

Other

责任方

Principal Investigator

主要研究者

Ercument Ovali

Specialist, Haematology

Acibadem University

研究点 (2)

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