A Registered Cohort Study on Duchenne Muscular Dystrophy

March 18, 2021 updated by: Ning Wang, MD., PhD.
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.

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Anticipated)

2000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Fuzhou, China
        • Recruiting
        • First Affiliated Hospital of Fujian Medical University
        • Contact:
        • Sub-Investigator:
          • Wan-Jin Chen, MD,PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients who are diagnosed as Duchenne Muscular Dystrophy in the hosipital

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Age at death
Time Frame: 20 years
the time when patient die
20 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ning Wang, MD., PhD.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2019

Primary Completion (Anticipated)

December 31, 2039

Study Completion (Anticipated)

December 31, 2049

Study Registration Dates

First Submitted

July 7, 2019

First Submitted That Met QC Criteria

July 7, 2019

First Posted (Actual)

July 9, 2019

Study Record Updates

Last Update Posted (Actual)

March 22, 2021

Last Update Submitted That Met QC Criteria

March 18, 2021

Last Verified

February 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MRCTA,ECFAH of FMU [2019]193

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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