A Registered Cohort Study on Duchenne Muscular Dystrophy

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04012671

Enrollment

2000

Registered

2019-07-09

Start date

2019-07-01

Completion date

2049-12-31

Last updated

2021-03-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Age at death	20 years	the time when patient die

Countries

China

Contacts

Primary ContactNing Wang, MD, PhD

ningwang@fjmu.edu.cn13805015340

Backup ContactMing Jin, MD

Safariday@live.com

Outcome results