241 Participants Needed

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

Recruiting at 21 trial locations
MI
ST
Overseen BySarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
Age: Any Age
Sex: Any
Trial Phase: Academic
Sponsor: Sarepta Therapeutics, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Who Is on the Research Team?

MD

Medical Director

Principal Investigator

Sarepta Therapeutics, Inc.

Are You a Good Fit for This Trial?

Inclusion Criteria

- Male or female participant ≥ 4 years of age with confirmed genetic diagnosis of LGMD2E/R4, LGMD2D/R3, or LGMD2C/R5.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Baseline Assessment

Participants undergo baseline assessments including genetic confirmation and NSAD scoring

Follow-up

Participants are monitored to evaluate mobility and pulmonary function

3 to 5 years

What Are the Treatments Tested in This Trial?

Interventions

  • No Investigational New Treatment

Find a Clinic Near You

Who Is Running the Clinical Trial?

Sarepta Therapeutics, Inc.

Lead Sponsor

Trials
54
Recruited
34,000+
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