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NCT03375255 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of Muscular Dystrophy, Duchenne, sponsored by Sarepta Therapeutics.

Completed
Registry status
Phase 1
Development phase
15
Enrollment target
8
Study locations

NCT03375255 is a Phase 1 study of Muscular Dystrophy, Duchenne that has completed, run by Sarepta Therapeutics. The registered enrollment target is 15 participants, below the 104-participant average among 4 other Muscular Dystrophy, Duchenne trials with a reported enrollment target (86% lower). The trial reports 8 study locations across 8 states.

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The verdict

NCT03375255, a Phase 1 study of Muscular Dystrophy, Duchenne, has completed, sponsored by Sarepta Therapeutics.

COMPLETED
Registry status
Phase 1
Development phase
15 participants
Enrollment target
8
Study locations

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 Studied

Muscular Dystrophy, Duchenne

Interventions

  • DRUG SRP-5051

Study Locations (8)

California

  • Neuromuscular Research Center - Sacramento

Florida

  • NW FL Clinical Research Group, LLC - Gulf Breeze

Georgia

  • Rare Disease Research, LLC - Atlanta

Illinois

  • Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago

Kansas

  • University of Kansas Medical Center - Kansas City

Pennsylvania

  • Children's Hospital of Pittsburgh of UPMC - Pittsburgh

Texas

  • Children's Medical Center Dallas - Dallas

Ontario

  • London Health Sciences Centre - London

Trial Details

FieldValue
Enrollment Target 15 participants
Start Date 2018-02-05
Est. Completion 2019-08-19
Phase Phase 1

Sponsor

Sarepta Therapeutics

33 total trials

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What the Registry Record Tells You About NCT03375255

The ClinicalTrials.gov registry entry for NCT03375255 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 104-participant average among 4 other Muscular Dystrophy, Duchenne trials with a reported enrollment target (86% lower). The listed sponsor is Sarepta Therapeutics, which has 33 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Muscular Dystrophy, Duchenne appearing as the primary indexed condition, and to 1 intervention - of which SRP-5051 is the first listed.

NCT03375255 reports 8 study locations spanning 8 distinct geographic areas - top geographies include California, Florida, Georgia.

Frequently Asked Questions

What is clinical trial NCT03375255 about?

NCT03375255 is a clinical study titled "A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)". 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.

What is the current status of trial NCT03375255?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2018-02-05. Estimated completion is 2019-08-19.

What conditions does trial NCT03375255 study?

This clinical trial studies the following conditions: Muscular Dystrophy, Duchenne.

What interventions are being tested in trial NCT03375255?

The interventions under investigation include: SRP-5051 (DRUG).

Who is sponsoring clinical trial NCT03375255?

This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT03375255 being conducted?

This trial has 8 study locations across California, Florida, Georgia, Illinois, Kansas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.