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NCT04475926ACTIVE 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

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
Limb-girdle Muscular Dystrophy
Interventions / Drugs
View on ClinicalTrials.gov ↗
Sponsor
SRPT
Sarepta Therapeutics
Gene Therapy
C54/100
Study Details
Enrollment205 participants
Primary Completion—
Lead SponsorSarepta Therapeutics, Inc.
Data Retrieved2026-04-11 02:55:56.327668+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 205 participants
Sponsor Catalyst Profile

Sarepta Therapeutics (SRPT) is the sponsoring company for this trial. BiotechSign currently grades this company C (54/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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