Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
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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
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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 or disease | Intervention/treatment | Phase |
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Duchenne Muscular Dystrophy | Biological: human umbilical cord mesenchymal stem cells | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 15 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy |
Study Start Date : | October 2011 |
Estimated Primary Completion Date : | March 2013 |
Estimated Study Completion Date : | October 2013 |

Arm | Intervention/treatment |
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Experimental: Intervention Group
Participants will be given rehabilitation therapy plus human umbilical cord mesenchymal stem cells transplantation with one year follow-up
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Biological: human umbilical cord mesenchymal stem cells
rehabilitation therapy plus human umbilical cord mesenchymal stem cells |
- Activities of Daily Living(ADL)scale [ Time Frame: 1 year after treatment ]
- 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 ]

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Ages Eligible for Study: | 5 Years to 12 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01610440
Contact: Liqing Yao | yaoliqing98731@yahoo.com.cn |
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 |
Principal Investigator: | Liqing Yao | The Second Affiliated Hospital of Kunming Medical University |
Responsible Party: | Shenzhen Beike Bio-Technology Co., Ltd. |
ClinicalTrials.gov Identifier: | NCT01610440 History of Changes |
Other Study ID Numbers: |
BKCR-DMD-1(Ⅰ) |
First Posted: | June 4, 2012 Key Record Dates |
Last Update Posted: | November 30, 2012 |
Last Verified: | November 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 |