NCT01610440

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Oct 2011

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2011

Completed
8 months until next milestone

First Submitted

Initial submission to the registry

May 21, 2012

Completed
14 days until next milestone

First Posted

Study publicly available on registry

June 4, 2012

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2013

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2013

Completed
Last Updated

November 30, 2012

Status Verified

November 1, 2012

Enrollment Period

1.4 years

First QC Date

May 21, 2012

Last Update Submit

November 28, 2012

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophyHuman Umbilical Cord Mesenchymal Stem Cells

Outcome Measures

Primary Outcomes (1)

  • Activities of Daily Living(ADL)scale

    1 year after treatment

Secondary Outcomes (7)

  • 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

  • +2 more secondary outcomes

Study Arms (1)

Intervention Group

EXPERIMENTAL

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

Interventions

human umbilical cord mesenchymal stem cellsBIOLOGICAL

rehabilitation therapy plus human umbilical cord mesenchymal stem cells

Intervention Group

Eligibility Criteria

Age5 Years - 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Second Affiliated Hospital of Kunming Medical College

Kunming, Yunnan, 650031, China

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Liqing Yao

    The Second Affiliated Hospital of Kunming Medical University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Liqing Yao

CONTACT

yaoliqing98731@yahoo.com.cn

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 21, 2012

First Posted

June 4, 2012

Study Start

October 1, 2011

Primary Completion

March 1, 2013

Study Completion

October 1, 2013

Last Updated

November 30, 2012

Record last verified: 2012-11

Locations

CN(1)