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

Primary Purpose

Duchenne Muscular Dystrophy

Status

Unknown status

Phase

Phase 1

Locations

China

Study Type

Interventional

Intervention

human umbilical cord mesenchymal stem cells

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne muscular dystrophy, Human Umbilical Cord Mesenchymal Stem Cells

Eligibility Criteria

5 Years - 12 Years (Child)All SexesDoes not accept 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

Sites / Locations

The Second Affiliated Hospital of Kunming Medical CollegeRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Intervention Group

Arm Description

Participants will be given rehabilitation therapy plus human umbilical cord mesenchymal stem cells transplantation with one year follow-up

Outcomes

Primary Outcome Measures

Activities of Daily Living(ADL)scale

Secondary Outcome Measures

Incidences of Adverse Event and Serious Adverse Event

Change from baseline in CK

Change from baseline in LDH

Change from baseline in ALT

Change from baseline in AST

Change from baseline to manual muscle test(MMT)

Change from baseline in electromyography(EMG)

Full Information

NCT ID

NCT01610440

First Posted

May 21, 2012

Last Updated

November 28, 2012

Sponsor

Shenzhen Beike Bio-Technology Co., Ltd.

Collaborators

The Second Affiliated Hospital of Kunming Medical University

1. Study Identification

Unique Protocol Identification Number

NCT01610440

Brief Title

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

2. Study Status

Record Verification Date

November 2012

Overall Recruitment Status

Unknown status

Study Start Date

October 2011 (undefined)

Primary Completion Date

March 2013 (Anticipated)

Study Completion Date

October 2013 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Shenzhen Beike Bio-Technology Co., Ltd.

Collaborators

The Second Affiliated Hospital of Kunming Medical University

4. Oversight

Data Monitoring Committee

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophy, Human Umbilical Cord Mesenchymal Stem Cells

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

15 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Intervention Group

Arm Type

Experimental

Arm Description

Participants will be given rehabilitation therapy plus human umbilical cord mesenchymal stem cells transplantation with one year follow-up

Intervention Type

Biological

Intervention Name(s)

human umbilical cord mesenchymal stem cells

Intervention Description

rehabilitation therapy plus human umbilical cord mesenchymal stem cells

Primary Outcome Measure Information:

Title

Activities of Daily Living(ADL)scale

Time Frame

1 year after treatment

Secondary Outcome Measure Information:

Title

Incidences of Adverse Event and Serious Adverse Event

Time Frame

1 year after treatment

Title

Change from baseline in CK

Time Frame

1 year after treatment

Title

Change from baseline in LDH

Time Frame

1 year after treatment

Title

Change from baseline in ALT

Time Frame

1 year after treatment

Title

Change from baseline in AST

Time Frame

1 year after treatment

Title

Change from baseline to manual muscle test(MMT)

Time Frame

1 year after treatment

Title

Change from baseline in electromyography(EMG)

Time Frame

1 year after treatment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Liqing Yao

yaoliqing98731@yahoo.com.cn

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Liqing Yao

Organizational Affiliation

The Second Affiliated Hospital of Kunming Medical University

Official's Role

Principal Investigator

Facility Information:

Facility Name

The Second Affiliated Hospital of Kunming Medical College

City

Kunming

State/Province

Yunnan

ZIP/Postal Code

650031

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Liqing Yao

yaoliqing98731@yahoo.com.cn

First Name & Middle Initial & Last Name & Degree

Liqing Yao

12. IPD Sharing Statement

Learn more about this trial

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

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