Clinical Research Study Opportunities

Are you or a family member with LGMD2A interested in participating in clinical research studies or trials? Below are some resources to help you learn about what clinical research studies are, why they matter, and what it means to participate.

National Institute of Health’s Clinical Research Trials and You

Office of Human Research Protection’s Research Participation Informational Videos

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The following studies are recruiting individuals with LGMD2A. If you know of other studies, please email C3 at info@curecalpain3.org so we can add them here. The links provided will bring you to a page which includes a detailed description of the study, eligibility criteria, study location(s), and contact information.

Trial Readiness and Endpoint Assessment in LGMD R1 (GRASP-01-003)

This observational study will follow LGMD2A/R1 patients over a period of 24 months to establish which Clinical Outcome Assessments are sensitive to normal disease progression, so that these may be used as tools during future clinical trials. Assessments include quantitative muscle MRI (qMR) of the upper and lower leg muscles to measure muscle fat fraction.

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD) (GRASP-001)

This observational study will follow LGMD2A/R1 patients over a period of 12 months to establish which Clinical Outcome Assessments are sensitive to normal disease progression, so that these may be used as tools during future clinical trials.

JOURNEY: A Global, Multicenter, Longitudinal Study of the Natural History of Subjects with limb-girdle muscular dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), Type 2C (LGMD2C/R5), and Type 2A (LGMD2A/R1)

This observational study will evaluate mobility and pulmonary function in individuals with LGMD2A/R1 over a period of up to three years

Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E

This is an observational study, no drug (marketed or investigational) will be provided as part of the study, and the study procedures will have no impact on the medical care delivered to patients participating in this study. The overall study data collection period is planned to last up to 5 years with assessments occurring at baseline, and every 6 months thereafter for a total period of 3 years.