To understand the role of clinical research studies in drug development, see Drug Development 101.
The following studies are enrolling individuals with LGMD2A/R1. If you know of other studies for LGMD2A/R1 or LGMDD4, 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) ACTIVE, not recruiting
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.
Evolution of the Functional and Muscular State of Patients With Limb-Girdle Muscular Dystrophy 2A (CALNATHIS) ACTIVE, not recruiting
This observational study will follow LGMD2A/R1 patients 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 affiliated with a social security scheme in France (beneficiary or entitled person).
Resources
Are you or a family member with LGMD2A/R1 or LGMDD4 interested in participating in clinical research studies or trials? Here is a resource to help you learn about what clinical research studies are, why they matter, and what it means to participate:
Office of Human Research Protection’s Research Participation Informational Videos