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

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

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.

Study Overview

• Status: Unknown
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Biological: human umbilical cord mesenchymal stem cells

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 Type: Interventional
• Enrollment (Anticipated): 15
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Liqing Yao; Email: yaoliqing98731@yahoo.com.cn

Study Locations

• China
  • Yunnan
    • Kunming, Yunnan, China, 650031
      • Recruiting
      • The Second Affiliated Hospital of Kunming Medical College
      • Contact: Liqing Yao; Email: yaoliqing98731@yahoo.com.cn
      • Principal Investigator: Liqing Yao

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Shenzhen Beike Bio-Technology Co., Ltd.
• Collaborators: The Second Affiliated Hospital of Kunming Medical University
• Investigators: Principal Investigator: Liqing Yao, The Second Affiliated hospital of Kunming Medical University

Study record dates

Study Major Dates

• Study Start: October 1, 2011
• Primary Completion (Anticipated): March 1, 2013
• Study Completion (Anticipated): October 1, 2013

Study Registration Dates

• First Submitted: May 21, 2012
• First Submitted That Met QC Criteria: June 1, 2012
• First Posted (Estimate): June 4, 2012

Study Record Updates

• Last Update Posted (Estimate): November 30, 2012
• Last Update Submitted That Met QC Criteria: November 28, 2012
• Last Verified: November 1, 2012

More Information

Terms related to this study

• Keywords: Duchenne muscular dystrophy; Human Umbilical Cord Mesenchymal Stem Cells
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: BKCR-DMD-1(Ⅰ)