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

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2012 by Shenzhen Beike Bio-Technology Co., Ltd.
Sponsor:
Collaborator:
The Second Affiliated Hospital of Kunming Medical University
Information provided by (Responsible Party):
Shenzhen Beike Bio-Technology Co., Ltd.
ClinicalTrials.gov Identifier:
NCT01610440
First received: May 21, 2012
Last updated: November 28, 2012
Last verified: November 2012
  Purpose

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.


Condition Intervention Phase
Duchenne Muscular Dystrophy
Biological: human umbilical cord mesenchymal stem cells
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy

Resource links provided by NLM:


Further study details as provided by Shenzhen Beike Bio-Technology Co., Ltd.:

Primary Outcome Measures:
  • Activities of Daily Living(ADL)scale [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Incidences of Adverse Event and Serious Adverse Event [ Time Frame: 1 year after treatment ] [ Designated as safety issue: Yes ]
  • Change from baseline in CK [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]
  • Change from baseline in LDH [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]
  • Change from baseline in ALT [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]
  • Change from baseline in AST [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]
  • Change from baseline to manual muscle test(MMT) [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]
  • Change from baseline in electromyography(EMG) [ Time Frame: 1 year after treatment ] [ Designated as safety issue: No ]

Estimated Enrollment: 15
Study Start Date: October 2011
Estimated Study Completion Date: October 2013
Estimated Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intervention Group
Participants will be given rehabilitation therapy plus human umbilical cord mesenchymal stem cells transplantation with one year follow-up
Biological: human umbilical cord mesenchymal stem cells
rehabilitation therapy plus human umbilical cord mesenchymal stem cells

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.

  Eligibility

Ages Eligible for Study:   5 Years to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01610440

Contacts
Contact: Liqing Yao yaoliqing98731@yahoo.com.cn

Locations
China, Yunnan
The Second Affiliated Hospital of Kunming Medical College Recruiting
Kunming, Yunnan, China, 650031
Contact: Liqing Yao       yaoliqing98731@yahoo.com.cn   
Principal Investigator: Liqing Yao         
Sponsors and Collaborators
Shenzhen Beike Bio-Technology Co., Ltd.
The Second Affiliated Hospital of Kunming Medical University
Investigators
Principal Investigator: Liqing Yao The Second Affiliated Hospital of Kunming Medical University
  More Information

No publications provided

Responsible Party: Shenzhen Beike Bio-Technology Co., Ltd.
ClinicalTrials.gov Identifier: NCT01610440     History of Changes
Other Study ID Numbers: BKCR-DMD-1(Ⅰ)
Study First Received: May 21, 2012
Last Updated: November 28, 2012
Health Authority: China: Ministry of Health

Keywords provided by Shenzhen Beike Bio-Technology Co., Ltd.:
Duchenne muscular dystrophy
Human Umbilical Cord Mesenchymal Stem Cells

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 23, 2014