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

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.

Verified November 2012 by Shenzhen Beike Bio-Technology Co., Ltd..
Recruitment status was: Recruiting

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

History of Changes

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:	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:

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy

MedlinePlus related topics: Muscular Dystrophy

Genetic and Rare Diseases Information Center resources: Muscular Dystrophy Duchenne Muscular Dystrophy Becker Muscular Dystrophy

U.S. FDA Resources

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 ]

Secondary Outcome Measures:

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 ]


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 (Child)
Sexes Eligible for Study:	All
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


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

Keywords provided by Shenzhen Beike Bio-Technology Co., Ltd.:


Duchenne muscular dystrophy Human Umbilical Cord Mesenchymal Stem Cells

Additional relevant MeSH terms:


Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases	Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on June 28, 2017

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