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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

NCT04004065 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 62

Last updated 2025-03-10

No results posted yet for this study

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

Interventions

DRUG

Vesleteplirsen

Vesleteplirsen injection, for IV use

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
7 Years
Max Age
21 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-06-26
Primary Completion
2023-10-30
Completion
2025-02-07
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • Germany
  • Italy
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04004065 on ClinicalTrials.gov