Are you or a family member with LGMD2A/R1 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
The following studies are enrolling individuals with LGMD2A/R1. 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.
This observational study will follow LGMD2A/R1 patients to establish to identify and quantify the loss of strength of the muscle groups of the upper and lower limbs over a 24 months. The participant must be an affiliate to a social security scheme in France (beneficiary or entitled person).
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. [ACTIVE, NOT RECRUITING]