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ACTIVE NOT RECRUITING

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

NCT04475926 · View on ClinicalTrials.gov ↗

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.

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

10 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04475926

The ClinicalTrials.gov registry entry for NCT04475926 describes a study currently listed as active not recruiting. It is categorized as an unspecified phase, 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 205 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 Limb-girdle Muscular Dystrophy appearing as the primary indexed condition, and to 0 interventions. 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: NCT04475926 reports 20 study locations spanning 19 distinct geographic areas — top geographies include California, Arizona, Arkansas. 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 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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

Who is sponsoring clinical trial NCT04475926?

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 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.

Related

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