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Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

key information

study id #: NCT04004065

condition: Duchenne Muscular Dystrophy

status: recruiting


This study will be comprised of 2 parts: Part A (Multiple Ascending Dose [MAD]) which will be conducted to evaluate the safety and tolerability of SRP-5051 at multiple ascending dose levels to determine the maximum tolerated dose (MTD); Part B (Dose Expansion) will be conducted to evaluate SRP-5051 administered at the MTD, both in patients who will complete Part A and in an expansion cohort of approximately 15 patients who will be enrolled in the study at the beginning of Part B.

intervention: SRP-5051

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT04004065

last updated: October 06, 2020