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

Sponsor

Shenzhen Beike Bio-Technology Co., Ltd. (Industry)

Overall Status

Unknown status

CT.gov ID

NCT01610440

Collaborator

The Second Affiliated Hospital of Kunming Medical University (Other)

Enrollment

Location

Arm

Duration (Months)

0.6

Patients Per Site Per Month

Study Details

Study Description

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.

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

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

15 participants

Allocation:

N/A

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 :

Oct 1, 2011

Anticipated Primary Completion Date :

Mar 1, 2013

Anticipated Study Completion Date :

Oct 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
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

Arm

Intervention/Treatment

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

Outcome Measures

Primary Outcome Measures

Activities of Daily Living(ADL)scale [1 year after treatment]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

5 Years to 12 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	The Second Affiliated Hospital of Kunming Medical College	Kunming	Yunnan	China	650031

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Shenzhen Beike Bio-Technology Co., Ltd.

ClinicalTrials.gov Identifier:

NCT01610440

Other Study ID Numbers:

BKCR-DMD-1(Ⅰ)

First Posted:

Jun 4, 2012

Last Update Posted:

Nov 30, 2012

Last Verified:

Nov 1, 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

Study Results

No Results Posted as of Nov 30, 2012