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A Registered Cohort Study on Duchenne Muscular Dystrophy

key information

study id #: NCT04012671

condition: Duchenne Muscular Dystrophy

status: recruiting

purpose:

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.

results: https://clinicaltrials.gov/ct2/show/results/NCT04012671

last updated: July 19, 2019

study details

start date: July 2019

estimated completion: December 31, 2039

phase of development: N/A

size / enrollment: 2000

primary outcomes:

  • Age at death [ Time Frame: 20 years ]
    The time when patient die

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 contacts

sponsor: Ning Wang, MD, PhD

contacts: Ning Wang (MD, PhD), 13805015340 ext 13805015340, ningwang@fjmu.edu.cn; Ming Jin MD, Safariday@live.com

locations: China