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A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant Children With DMD

key information

study id #: NCT01761292

condition: Duchenne Muscular Dystrophy (DMD)

status: completed


This is a 2-part, phase 2 study to assess the effects of Givinostat on muscle histologic parameters and on clinical parameters in ambulant children with DMD. The safety, tolerability, and pharmacokinetics of Givinostat will also be assessed. All children treated in part 2 continue in the extension phase, for a maximum of an additional 12 months.

intervention: Givinostat

mechanism of action: Histone deacetylase (HDAC) inhibitor to reduce inflammation and promote muscle health

results: https://clinicaltrials.gov/ct2/show/results/NCT01761292

last updated: November 22, 2018