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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01610440
Recruitment Status : Unknown
Verified November 2012 by Shenzhen Beike Bio-Technology Co., Ltd..
Recruitment status was:  Recruiting
First Posted : June 4, 2012
Last Update Posted : November 30, 2012
Sponsor:
Collaborator:
The Second Affiliated Hospital of Kunming Medical University
Information provided by (Responsible Party):
Shenzhen Beike Bio-Technology Co., Ltd.

Tracking Information
First Submitted Date  ICMJE May 21, 2012
First Posted Date  ICMJE June 4, 2012
Last Update Posted Date November 30, 2012
Study Start Date  ICMJE October 2011
Estimated Primary Completion Date March 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 1, 2012)
Activities of Daily Living(ADL)scale [ Time Frame: 1 year after treatment ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 28, 2012)
  • 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 Measures  ICMJE
 (submitted: June 1, 2012)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE 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 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Biological: human umbilical cord mesenchymal stem cells
rehabilitation therapy plus human umbilical cord mesenchymal stem cells
Study Arms  ICMJE Experimental: Intervention Group
Participants will be given rehabilitation therapy plus human umbilical cord mesenchymal stem cells transplantation with one year follow-up
Intervention: Biological: human umbilical cord mesenchymal stem cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: June 1, 2012)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2013
Estimated Primary Completion Date March 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Aged 5-12 years
  • Clinical manifestation, enzymology, electromyogram, gene type confirmed the diagnose of Duchenne muscular dystrophy
  • Sign the consent form and follow the clinic trail procedure

Exclusion Criteria:

  • Not Duchenne muscular dystrophy
  • Any history of hypersensitivity to serum products,or other know drug and food allergy
  • Combined Pneumonia or other Severe systemic bacteria infection
  • HIV+, TPPA +, patients diagnosed as HBV or HCV
  • Tumor Markers +
  • Severe psychotic patients, cognitive dysfunction
  • Coagulation disorders
  • Uncontrolled hypertension after treatment,blood pressure≥180mmHg/110 mmHg
  • Other severe systemic or organic disease
  • Enrollment in other trials in the last 3 months
  • Received any stem cell therapy in past 6 months
  • Other criteria that investigator consider improper for inclusion
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01610440
Other Study ID Numbers  ICMJE BKCR-DMD-1(Ⅰ)
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Shenzhen Beike Bio-Technology Co., Ltd.
Study Sponsor  ICMJE Shenzhen Beike Bio-Technology Co., Ltd.
Collaborators  ICMJE The Second Affiliated Hospital of Kunming Medical University
Investigators  ICMJE
Principal Investigator: Liqing Yao The Second Affiliated Hospital of Kunming Medical University
PRS Account Shenzhen Beike Bio-Technology Co., Ltd.
Verification Date November 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP