Overview

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

Status:
Active, not recruiting
Trial end date:
2022-05-31
Target enrollment:
Participant gender:
Summary
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of SRP-5051 at multiple ascending dose levels to determine the maximum tolerated dose (MTD), and 2) Part B (Dose Expansion) will be conducted to evaluate SRP-5051 administered at the MTD determined in Part A or, if applicable, at a second dose introduced in Part B. Patients enrolling in Part B will be those who completed Part A as well as additional expansion cohort patients who will enroll at the beginning of Part B.
Phase:
Phase 2
Details
Lead Sponsor:
Sarepta Therapeutics
Sarepta Therapeutics, Inc.