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

Study Status: Withdrawn
Recruit Status: Unknown status
Study Type: Interventional

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

Brief 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 Summary: This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive muscular dystrophy.
Sponsor: Shenzhen Beike Bio-Technology Co., Ltd.

Current Primary Outcome: Activities of Daily Living(ADL)scale [ Time Frame: 1 year after treatment ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Incidences of Adverse Event and Serious Adverse Event [ Time Frame: 1 year after treatment ]
  • Change from baseline in CK [ Time Frame: 1 year after treatment ]
  • Change from baseline in LDH [ Time Frame: 1 year after treatment ]
  • Change from baseline in ALT [ Time Frame: 1 year after treatment ]
  • Change from baseline in AST [ Time Frame: 1 year after treatment ]
  • Change from baseline to manual muscle test(MMT) [ Time Frame: 1 year after treatment ]
  • Change from baseline in electromyography(EMG) [ Time Frame: 1 year after treatment ]


Original Secondary Outcome:

  • Incidences of Adverse Event and Serious Adverse Event [ Time Frame: 1 year after treatment ]
  • Change from baseline in CK [ Time Frame: Month 0, 3, 6, 9, 12 ]
  • Change from baseline in LDH [ Time Frame: Month 0, 3, 6, 9, 12 ]
  • Change from baseline in ALT [ Time Frame: Month 0, 3, 6, 9, 12 ]
  • Change from baseline in AST [ Time Frame: Month 0, 3, 6, 9, 12 ]
  • Change from baseline to manual muscle test(MMT) [ Time Frame: Month 0, 3, 6, 9, 12 ]
  • Change from baseline in electromyography(EMG) [ Time Frame: Month 0, 3, 6, 9, 12 ]


Information By: Shenzhen Beike Bio-Technology Co., Ltd.

Dates:
Date Received: May 21, 2012
Date Started: October 2011
Date Completion: October 2013
Last Updated: November 28, 2012
Last Verified: November 2012