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A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

NCT03375255 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2022-07-06

No results posted yet for this study

Summary

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 (vesleteplirsen) administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.

Conditions

  • Muscular Dystrophy, Duchenne

Interventions

DRUG

SRP-5051

Single dose of SRP-5051 administered as an intravenous (IV) infusion.

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-02-05
Primary Completion
2019-08-19
Completion
2019-08-19
FDA Drug
Yes

Countries

  • United States
  • Canada

Study Locations

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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 NCT03375255 on ClinicalTrials.gov