Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 57 of 1005 for:    BMD

A Registered Cohort Study on Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04012671
Recruitment Status : Recruiting
First Posted : July 9, 2019
Last Update Posted : September 23, 2019
Sponsor:
Information provided by (Responsible Party):
Ning Wang, MD., PhD., First Affiliated Hospital of Fujian Medical University

Brief Summary:
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy to optimize clinical management.

Condition or disease
Duchenne Muscular Dystrophy

Layout table for study information
Study Type : Observational
Estimated Enrollment : 2000 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Registered Cohort Study on Duchenne Muscular Dystrophy
Actual Study Start Date : July 1, 2019
Estimated Primary Completion Date : December 31, 2039
Estimated Study Completion Date : December 31, 2049





Primary Outcome Measures :
  1. Age at death [ Time Frame: 20 years ]
    the time when patient die



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients who are diagnosed as Duchenne Muscular Dystrophy in the hosipital
Criteria

Inclusion Criteria:

  • Beyond 2 years old
  • Diagnosis with Duchenne Muscular Dystrophy, and female carriers, genotypically confirmed
  • Diagnosis should be supported by muscle biopsy, if no genetic confirmation.

Exclusion Criteria:

  • Presence of other clinically significant illness

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04012671


Contacts
Layout table for location contacts
Contact: Ning Wang, MD, PhD 13805015340 ext 13805015340 ningwang@fjmu.edu.cn
Contact: Ming Jin, MD Safariday@live.com

Locations
Layout table for location information
China
First Affiliated Hospital of Fujian Medical University Recruiting
Fuzhou, China
Contact: Ming Jin, MD       Safariday@live.com   
Principal Investigator: Ning Wang, MD,PhD         
Sub-Investigator: Wan-Jin Chen, MD,PhD         
Sponsors and Collaborators
Ning Wang, MD., PhD.

Layout table for additonal information
Responsible Party: Ning Wang, MD., PhD., Professor, First Affiliated Hospital of Fujian Medical University
ClinicalTrials.gov Identifier: NCT04012671     History of Changes
Other Study ID Numbers: MRCTA,ECFAH of FMU [2019]193
First Posted: July 9, 2019    Key Record Dates
Last Update Posted: September 23, 2019
Last Verified: September 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked