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COMPLETED 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)

NCT03375255 · View on ClinicalTrials.gov ↗

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.

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

10 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03375255

The ClinicalTrials.gov registry entry for NCT03375255 describes a study currently listed as completed. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sarepta Therapeutics, which has 10 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT03375255 reports 8 study locations spanning 8 distinct geographic areas — top geographies include California, Florida, Georgia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT03375255?

The interventions under investigation include: SRP-5051 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03375255?

This trial is sponsored by Sarepta Therapeutics, which has 10 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial