Status:
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
Lead Sponsor:
Sarepta Therapeutics, Inc.
Conditions:
Limb-girdle Muscular Dystrophy
Eligibility:
All Genders
4+ years
Brief 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), L...
Detailed Description
LGMD Type 2A, 2C, and 2D cohorts are now closed. Enrollment in the LGMD Type 2E cohort is closed and participants will be followed for up to five years.
Eligibility Criteria
Inclusion
- Male or female participant ≥ 4 years of age who demonstrate symptoms of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1 in the opinion of the investigator (eg, muscle weakness, loss of function, delayed milestones).
- Confirmed clinical and genetic diagnosis of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1.
Exclusion
- Demonstrates cognitive delay or impairment that could confound motor development, in the opinion of the Investigator.
- Has a medical condition, in the opinion of the Investigator, that might compromise participants ability to comply with study requirements.
- Is participating in other interventional study(ies) at the time of enrollment in this study.
Key Trial Info
Start Date :
April 22 2021
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 5 2030
Estimated Enrollment :
205 Patients enrolled
Trial Details
Trial ID
NCT04475926
Start Date
April 22 2021
End Date
September 5 2030
Last Update
November 6 2025
Active Locations (26)
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1
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
2
Arkansas Children's
Little Rock, Arkansas, United States, 72202
3
University of California San Diego
La Jolla, California, United States, 92037
4
University of California, Davis Health Dept of PM&R
Sacramento, California, United States, 95817