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

Study Overview
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Brief Summary
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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.

Detailed Description
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This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive muscular dystrophy.

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

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Biological: human umbilical cord mesenchymal stem cells


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intervention Group<br>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<br>* rehabilitation therapy plus human umbilical cord mesenchymal stem cells<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Activities of Daily Living(ADL)scale |  | 1 year after treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidences of Adverse Event and Serious Adverse Event |  | 1 year after treatment | 
| Change from baseline in CK |  | 1 year after treatment | 
| Change from baseline in LDH |  | 1 year after treatment | 
| Change from baseline in ALT |  | 1 year after treatment | 
| Change from baseline in AST |  | 1 year after treatment | 
| Change from baseline to manual muscle test(MMT) |  | 1 year after treatment | 
| Change from baseline in electromyography(EMG) |  | 1 year after treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Duchenne muscular dystrophy, Human Umbilical Cord Mesenchymal Stem Cells