Clinical trial NCT01610440: Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
Official title: Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne muscular dystrophy
Human Umbilical Cord Mesenchymal Stem Cells
Lead sponsor: Shenzhen Beike Bio-Technology Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: The Second Affiliated Hospital of Kunming Medical University
First posted: 2012-06-04
Enrollment: 15
Sex: ALL
Minimum age (years): 5
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Activities of Daily Living(ADL)scale
timeFrame: 1 year after treatment
Secondary outcomes:
measure: Incidences of Adverse Event and Serious Adverse Event
timeFrame: 1 year after treatment
measure: Change from baseline in CK
timeFrame: 1 year after treatment
measure: Change from baseline in LDH
timeFrame: 1 year after treatment
measure: Change from baseline in ALT
timeFrame: 1 year after treatment
measure: Change from baseline in AST
timeFrame: 1 year after treatment
measure: Change from baseline to manual muscle test(MMT)
timeFrame: 1 year after treatment
measure: Change from baseline in electromyography(EMG)
timeFrame: 1 year after treatment
Other outcomes:
Locations:
city: Kunming
country: China
facility: The Second Affiliated Hospital of Kunming Medical College
state: Yunnan
Study officials:
affiliation: The Second Affiliated Hospital of Kunming Medical University
name: Liqing Yao
role: PRINCIPAL_INVESTIGATOR
References: