A Registered Cohort Study on Duchenne Muscular Dystrophy

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.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

First Affiliated Hospital of Fujian Medical University

Fuzhou, China

Location status: Recruiting

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

Age at death

Time frame: 20 years

the time when patient die

Interested in participating?

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Sponsors and collaborators

Lead sponsor

Ning Wang, MD., PhD.

Other

Registry information

Important dates

Study start: 2019
Primary completion: 2039
Study completion: 2049
First posted: Jul 9, 2019
Registry last updated: Mar 22, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

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