The GRASP-LGMD Consortium is a group of doctors, physical therapists, and clinical research coordinators who collaborate to measure how the symptoms of LGMD change over time. Studies like this, called Natural History Studies, are critical for the design of future clinical trials. The results of the Consortium’s LGMD2A/R1 study were recently published in the journal Neuromuscular Disorders. This important work was supported by grants from C3, the Muscular Dystrophy Association, and the National Institutes for Health.
There are multiple tests to measure changes over one year
The study used several tests, called Clinical Outcome Assessments (COAs), to measure muscle function at the start, middle, and end of one year. Four COAs, all commonly used in clinical trials, were able to detect declines:
- North Star Assessment for LGMDs (NSAD): 29 items related to tasks of daily life, including lifting arms, rolling over, standing up from a chair, and walking
- Performance of Upper Limb (PUL): how well arms and hands function
- 100 Meter Timed Test (100MTT): how fast someone can walk or run 100 meters
- 4-Stair Climb (4SC): time to climb 4 steps
Planning for the future
The identification of multiple COAs is good news for future clinical trials, since it allows the selection of the most relevant COA for the participants of that trail. For example, the 100MTT and 4SC can only be completed by individuals who can walk independently, but both ambulatory and non-ambulatory individuals can perform the NSAD and PUL. Further, the study shows that a one year trial may be long enough to see if a new treatment is working in future clinical trials.
A big THANK YOU
We at C3 are grateful to the LGMD2A/R1 community members who participated in this work, as well as the Consortium members who performed the research. The COAs identified in the study, and the accompanying natural history data, serve as a “toolkit” for trial design, enhancing the likelihood of success when testing future treatments.