LGMD2A/R1 Patient Enrollment Underway for C3-Sponsored Natural History Study

The GRASP-LGMD consortium is currently recruiting for a natural history study for individuals with mutations in CAPN3 (calpain 3). This study will inform the design of future therapeutic trials and a better understanding of the disease. Participants can now enroll at Virginia Commonwealth University, Kansas University Medical Center, Washington University in St. Louis, University of California, Irvine, and University of Iowa. Participants must be 6-65 years old and ambulatory at the time of enrollment. Initial visits may be performed remotely, with later visits at 6 and 18 months occurring in person. Participants will be responsible for their own travel and lodging expenditures if applicable.

The primary purposes of this study are:

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• to define a common set of pelvic and shoulder girdle COAs* for future research
• to follow the progression of the disease

* What are COAs, and why do they matter to those living with LGMD? Clinical outcome assessments (COAs) are measurements used during clinical trials to determine if an investigational drug or therapy provides benefits. As therapeutics are being developed for limb girdle muscular dystrophies (LGMDs), it is important to prepare for clinical trials by developing robust COAs that are sensitive to changes and represent aspects of the disease that are important to patients. 

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INTERESTED IN PARTICIPATING? QUESTIONS?

Please contact tammy.carrington@vcuhealth.org