NCT04004065

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 7 Years – 21 Years
• Healthy volunteers: Not accepted

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 vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 (NCT03675126) and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Vesleteplirsen	Vesleteplirsen injection, for IV use

Timeline

• Start date: 2019-06-26
• Primary completion: 2023-10-30
• Completion: 2025-02-07
• First posted: 2019-07-01
• Last updated: 2025-03-10

Locations

25 sites across 8 countries: United States, Belgium, Canada, Germany, Italy, Netherlands, Spain, United Kingdom

Regulatory

• FDA-regulated drug study