Clinical Trial

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

Active, Not Recruiting
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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.
Protocol Amendment History 19 amendments
This ClinicalTrials.gov record has been amended 19 times since 2020-07-14; most recent amendment 2025-11-04.
Status change: Recruiting → Active, Not Recruiting 2025-11-04
Status change: Not Yet Recruiting → Recruiting 2020-09-23
Trial Details
NCT Number NCT04475926
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Limb-girdle Muscular Dystrophy
Enrollment 205 participants
Start Date 2021-04-22
Primary Completion 2030-09-05 (estimated)
Study Completion 2030-09-05 (estimated)
Updated on ClinicalTrials.gov 2025-11-06