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A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

key information

study id #: NCT03760029

condition: Duchenne Muscular Dystrophy

status: recruiting


This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients. A total of approximately 330 subjects will be enrolled with the target number of subjects in each group as below:

• Group 1, subjects aged <6 years, 100 subjects;

• Group 2, subjects aged >=6 years and <12 years, 180 subjects;

• Group 3, subjects aged >=12 years, 50 subjects.

Subjects will visit sites every 6 months. Each subject will be observed for at least 24 months. All subjects will remain enrolled until the study completion date, such that some will have data collected after Month 24. Subjects, who complete Visit 5/Month 24 at least 6 months prior to study completion, will be asked to complete an additional visit at Month 30.

intervention: Visit frequency

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT03760029

last updated: August 26, 2020