NCT01610440

Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy

Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Shenzhen Beike Bio-Technology Co., Ltd. · Industry
• Sex: All
• Age: 5 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive muscular dystrophy.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
BIOLOGICAL	human umbilical cord mesenchymal stem cells	rehabilitation therapy plus human umbilical cord mesenchymal stem cells

Timeline

• Start date: 2011-10-01
• Primary completion: 2013-03-01
• Completion: 2013-10-01
• First posted: 2012-06-04
• Last updated: 2012-11-30

Locations

1 site across 1 country: China