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COMPLETED Phase 1

A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT03375164 · View on ClinicalTrials.gov ↗

Study Summary

This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.

Interventions

  • GENETIC delandistrogene moxeparvovec

Study Locations (1)

Ohio

  • Nationwide Children's Hospital — Columbus

Trial Details

FieldValue
Enrollment Target 4 participants
Start Date 2018-01-04
Est. Completion 2023-04-25
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 NCT03375164

The ClinicalTrials.gov registry entry for NCT03375164 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 4 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 Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention — of which delandistrogene moxeparvovec 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: NCT03375164 reports 1 study location spanning 1 distinct geographic area — top geographies include Ohio. 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 NCT03375164 about?

NCT03375164 is a clinical study titled "A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)". This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3...

What is the current status of trial NCT03375164?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2018-01-04. Estimated completion is 2023-04-25.

What conditions does trial NCT03375164 study?

This clinical trial studies the following conditions: Duchenne Muscular Dystrophy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT03375164?

The interventions under investigation include: delandistrogene moxeparvovec (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03375164?

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 NCT03375164 being conducted?

This trial has 1 study location across Ohio. 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