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NCT06246513 · ClinicalTrials.gov registry record · Phase 3

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

A Phase 3 study of Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics.

Active
Registry status
Phase 3
Development phase
17
Enrollment target
10
Study locations

NCT06246513 is a Phase 3 study of Limb Girdle Muscular Dystrophy that is active but no longer recruiting, run by Sarepta Therapeutics. The registered enrollment target is 17 participants, below the 237-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target (93% lower). The trial reports 10 study locations across 7 states.

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The verdict

NCT06246513, a Phase 3 study of Limb Girdle Muscular Dystrophy, is active but no longer recruiting, sponsored by Sarepta Therapeutics.

ACTIVE NOT RECRUITING
Registry status
Phase 3
Development phase
17 participants
Enrollment target
10
Study locations

Study Summary

This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).

Conditions Studied

Limb Girdle Muscular Dystrophy

Interventions

  • DRUG Glucocorticoid
  • BIOLOGICAL SRP-9003

Study Locations (10)

Other

  • NMRC Gent (UZ Gent) - Ghent
  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico - Milan
  • Hospital Sant Joan de Deu - Barcelona
  • Newcastle University - Newcastle upon Tyne

California

  • University of California, San Diego-Altman Clinical and Translational Research Institute - La Jolla

Ohio

  • Nationwide Childrens Hospital - Columbus

Pennsylvania

  • The Children's Hospital of Philadelphia - Philadelphia

Virginia

  • Children's Hospital of The King's Daughter - Norfolk

Vlaams Brabant

  • University Hospital Leuven (UZ Leuven) - Leuven

North Rhine-Westphalia

  • Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum - Essen

Trial Details

FieldValue
Enrollment Target 17 participants
Start Date 2024-01-15
Est. Completion 2029-11-30
Phase Phase 3

Sponsor

Sarepta Therapeutics

33 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06246513

The ClinicalTrials.gov registry entry for NCT06246513 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 17 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 237-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target (93% lower). The listed sponsor is Sarepta Therapeutics, which has 33 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Limb Girdle Muscular Dystrophy appearing as the primary indexed condition, and to 2 interventions - of which Glucocorticoid is the first listed.

NCT06246513 reports 10 study locations spanning 7 distinct geographic areas - top geographies include Other, California, Ohio.

Frequently Asked Questions

What is clinical trial NCT06246513 about?

NCT06246513 is a clinical study titled "A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4". This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory parti...

What is the current status of trial NCT06246513?

This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 17 participants. The study started on 2024-01-15. Estimated completion is 2029-11-30.

What conditions does trial NCT06246513 study?

This clinical trial studies the following conditions: Limb Girdle Muscular Dystrophy.

What interventions are being tested in trial NCT06246513?

The interventions under investigation include: Glucocorticoid (DRUG), SRP-9003 (BIOLOGICAL).

Who is sponsoring clinical trial NCT06246513?

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

Where is trial NCT06246513 being conducted?

This trial has 10 study locations across California, Ohio, Pennsylvania, Virginia, Vlaams Brabant. 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.