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NCT04475926 · ClinicalTrials.gov registry record
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
A clinical trial of Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics.
- Active
- Registry status
- 205
- Enrollment target
- 20
- Study locations
NCT04475926: Active study of Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics.
NCT04475926 is a study of Limb Girdle Muscular Dystrophy that is active but no longer recruiting, run by Sarepta Therapeutics. The registered enrollment target is 205 participants, roughly in line with the 206-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target. The trial reports 20 study locations across 19 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04475926, a study of Limb Girdle Muscular Dystrophy, is active but no longer recruiting, sponsored by Sarepta Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- 205 participants
- Enrollment target
- 20
- Study locations
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 Studied
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
| Field | Value |
|---|---|
| Enrollment Target | 205 participants |
| Start Date | 2021-04-22 |
| Est. Completion | 2030-09-05 |
What the registry record for NCT04475926 still lists
NCT04475926 is an observational study that tracks outcomes without assigning an intervention. The registered 205 participants enrollment target is mid-sized for trials with a published cap, roughly in line with the 206-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target.
The record links to 1 condition, with Limb Girdle Muscular Dystrophy appearing as the primary indexed condition, and to 0 interventions.
NCT04475926 names 20 study sites across 19 states, led by California, Arizona, Arkansas.
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.
Who is sponsoring clinical trial NCT04475926?
This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.
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.
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