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Tamoxifen in Duchenne Muscular Dystrophy

key information

study id #: NCT03354039

condition: Duchenne Muscular Dystrophy

status: recruiting


A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne’s muscular dystrophy (DMD) patients that are under stable standard treatment of care with glucocorticoids. Furthermore, the investigators plan to include 6-20 non-ambulant patients who do not receive glucocorticoids (as parallel group B), 10 to 16 years old, to obtain efficacy and safety data in a broader DMD population. All patients will receive 20 mg of tamoxifen (TAM) or placebo once daily during 48 weeks.

intervention: Tamoxifen, Matching placebo

mechanism of action: Estrogen receptor antagonist to improve muslce strength

results: https://clinicaltrials.gov/ct2/show/results/NCT03354039

last updated: August 26, 2020