Clinical outcome assessments (COAs) are measurements used during clinical trials to determine if an investigational drug or therapy provides benefit. 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.

Dr. Johnson and colleagues recently established a Limb Girdle Muscular Dystrophy Clinical Research Network, funded by a grant from the Muscular Dystrophy Association, which brings together eight medical centers with significant expertise in LGMD research and clinical care. The current observational study will follow LGMD2A patients over a period of 12 months to establish which COAs are sensitive to normal disease progression, so that these may be used as tools during future clinical trials.

C3 Scientific Director Dr. Jennifer Levy explains, “A lack of standard COAs for LGMD2A is a barrier to therapy development for this disease. Dr. Johnson’s project will develop tools which will be used to determine if investigational drugs are effective in a clinical trial setting.”

Dr. Johnson notes, “The development of gene replacement therapies has accelerated the need to prepare for clinical trials. We are excited to begin working with C3, the MDA, and the LGMD2A community to develop the tools necessary to test these potential treatments.”