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NCT04475926 · ClinicalTrials.gov registry record

A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

A clinical trial of Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics.

Active
Registry status
205
Enrollment target
20
Study locations

NCT04475926 is a study of Limb Girdle Muscular Dystrophy that is active but no longer recruiting, run by Sarepta Therapeutics. The registered enrollment target is 205 participants, roughly in line with the 206-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target. The trial reports 20 study locations across 19 states.

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The verdict

NCT04475926, a study of Limb Girdle Muscular Dystrophy, is active but no longer recruiting, sponsored by Sarepta Therapeutics.

ACTIVE NOT RECRUITING
Registry status
205 participants
Enrollment target
20
Study locations

Study Summary

This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy type 2C (LGMD2C/R5), or Limb-girdle muscular dystrophy type 2A (LGMD2A/R1). These enrolled participants will be followed to evaluate mobility and pulmonary function for up to 5 years after enrollment for participants with LGMD2C/R5, LGMD2D/R3, and LGMD2E/R4 with a North Star Assessment for Dysferlinopathy (NSAD) ≥ 25 at Baseline, up to 3 years for participants with LGMD2C/R5, LGMD2D/R3, and LGMD2E/R4 with a NSAD \< 25 at Baseline, and up to 3 years for participants with LGMD2A/R1. Additional participant data will be collected from the time the individual began experiencing LGMD symptoms to the present.

Conditions Studied

Limb Girdle Muscular Dystrophy

Study Locations (20)

California

  • University of California San Diego - La Jolla
  • University of California, Davis Health Dept of PM&R - Sacramento

Arizona

  • Barrow Neurological Institute - Phoenix

Arkansas

  • Arkansas Children's - Little Rock

Illinois

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

Ohio

  • Nationwide Children's Hospital - Columbus

Oregon

  • Oregon Health and Science University - Portland

Pennsylvania

  • The Children's Hospital of Philadelphia - Philadelphia

Texas

  • University of Texas Southwestern Medical Center - Dallas

Trial Details

FieldValue
Enrollment Target 205 participants
Start Date 2021-04-22
Est. Completion 2030-09-05

Sponsor

Sarepta Therapeutics

33 total trials

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What the Registry Record Tells You About NCT04475926

The ClinicalTrials.gov registry entry for NCT04475926 describes a study currently listed as active not recruiting, categorized as an unspecified phase. The registered enrollment target is 205 participants, a figure that helps gauge the scale of data the investigators plan to collect, roughly in line with the 206-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target. 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 0 interventions.

NCT04475926 reports 20 study locations spanning 19 distinct geographic areas - top geographies include California, Arizona, Arkansas.

Frequently Asked Questions

What is clinical trial NCT04475926 about?

NCT04475926 is a clinical study titled "A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice". This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy type 2C (LGMD2C/R5), or Limb-girdle muscular dystrophy type 2A (LGMD2A...

What is the current status of trial NCT04475926?

This trial is currently active not recruiting. The enrollment target is 205 participants. The study started on 2021-04-22. Estimated completion is 2030-09-05.

What conditions does trial NCT04475926 study?

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

Who is sponsoring clinical trial NCT04475926?

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

Where is trial NCT04475926 being conducted?

This trial has 20 study locations across Arizona, Arkansas, California, Illinois, Ohio. 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.