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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01610440
• Other study ID #: BKCR-DMD-1(?)
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: May 21, 2012
• Last updated: November 28, 2012
• Start date: October 2011
• Est. completion date: October 2013

Study information

• Verified date: November 2012
• Source: Shenzhen Beike Bio-Technology Co., Ltd.
• Contact: Liqing Yao
• Email: yaoliqing98731[@]yahoo.com.cn
• Is FDA regulated: No
• Health authority: China: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: October 2013
• Est. primary completion date: March 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years to 12 Years

Eligibility

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 Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Biological:
• human umbilical cord mesenchymal stem cells
rehabilitation therapy plus human umbilical cord mesenchymal stem cells

Locations

Country	Name	City	State
China	The Second Affiliated Hospital of Kunming Medical College	Kunming	Yunnan

Sponsors (2)

Lead Sponsor	Collaborator
Shenzhen Beike Bio-Technology Co., Ltd.	The Second Affiliated Hospital of Kunming Medical University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Activities of Daily Living(ADL)scale		1 year after treatment	No
Secondary	Incidences of Adverse Event and Serious Adverse Event		1 year after treatment	Yes
Secondary	Change from baseline in CK		1 year after treatment	No
Secondary	Change from baseline in LDH		1 year after treatment	No
Secondary	Change from baseline in ALT		1 year after treatment	No
Secondary	Change from baseline in AST		1 year after treatment	No
Secondary	Change from baseline to manual muscle test(MMT)		1 year after treatment	No
Secondary	Change from baseline in electromyography(EMG)		1 year after treatment	No