Duchenne Muscular Dystrophy Clinical Trial
Official title:
Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed
slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as
impaired respiratory muscle and cardiac muscle. To a large extent, patients always lose
motor function gradually and die for heart failure or severe infection at the end stage of
DMD. At present, the treatment strategy relies on heteropathy accompanied with
rehabilitation training. However, the therapeutic effect remains extremely limited.
Human umbilical cord mesenchymal stem cells (hUC-MSCs) have been evidenced to improve motor
function, increase muscle strength and reduce abnormal levels of related enzymes, such as
creatine kinase (CK), lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and
aspartate aminotransferase (AST). This study is aimed to explore the safety and efficacy of
hUC-MSCs transplantation for DMD.
This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive muscular dystrophy. ;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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