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
← catalyst calendarNCT04475926 · readout in 1,478 d
Sponsored by Sarepta Therapeutics, Inc. (industry) · SRPT — their whole pipeline →.
Phase
—
Status
Active, not recruiting
Study type
Observational
Enrollment
205actual
Sites
26
Countries
Belgium, Brazil, Canada +7
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2021-04-22 | actual | When the study began enrolling |
| Primary completion | 2030-09-05 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2030-09-05 | estimated | The whole study's end, after follow-up |
| First posted | 2020-07-17 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-11-06 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Limb-girdle Muscular Dystrophy
Primary outcomes
what the primary-completion date above is the date OFNSAD Total Score
measured Baseline up to Month 60
Time to Rise from the Floor
measured Baseline up to Month 60
Time of 10-Meter Walk/Run [10MWR]
measured Baseline up to Month 60
Time to Ascend 4 Steps
measured Baseline up to Month 60
Ankle Range of Motion (ROM)
measured Baseline up to Month 60
Dimension of the Performance of the Upper Limb (PUL)
measured Baseline up to Month 60
Timed Up and Go (TUG)
measured Baseline up to Month 60
Time of 100-Meter Walk/Run (100MWR)
measured Baseline up to Month 60
Pulmonary Function Test: Forced Vital Capacity (FVC)
measured Baseline up to Month 60
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