Casimir Recruiting LGMD2A/R1 Patients for At-Home Study

The Casimir LGVA study is quickly filling! While the study is open to individuals of all abilities, the cohorts for those with more severe symptoms are nearly full. We are specifically seeking individuals with LGMD2A who can walk across a room and stand up from sitting on a couch to participate. You should be able to do these tasks without the assistance of another person, the use of an assistive device (such as a cane or walker), holding onto a wall or piece of furniture, rocking to gain momentum, or trying several times on a regular basis. The study can be done entirely from your home, and compensation is provided. Email LGMDS@casimirtrials.com or call (800)542-0948 for more information.

---

With the development of potential new avenues of treatment for Limb-Girdle Muscular Dystrophies (LGMDs) comes clinical trials to test the efficacy of gene therapy, gene editing, and other possibly revolutionary treatments for LGMDs. As these potential treatments and subsequent trials move forward, a sensitive and reliable, inclusive, and patient-centered outcome measure is needed to determine the benefits of the investigational treatments. Current outcome measures used in clinical trials for LGMDs are often not disease-specific and make use of timed function tests and clinician-rated assessments of movement compensations. These outcome measures often restrict participation to those with a high enough level of physical functioning to complete the tests and show change. In addition, clinical trials are conducted through a local site that limits participants to a geographical area and can create a significant travel burden for those who wish to be involved.

The Limb-Girdle Video Assessment (LGVA) is a proposed outcome measure being developed by Casimir specifically for use in LGMDs clinical trials and supported and co-funded by Coalition to Cure Calpain 3 and the Jain Foundation. We are developing the LGVA after the success and validation of the Duchenne Video Assessment (DVA), a home-based, patient-centered functional outcome measure for the Duchenne Muscular Dystrophy community. The goal of the LGVA study is to mimic the success of the DVA in evaluating a participant’s ease of movement in performing everyday tasks (e.g., getting in/out of a chair, moving around your home, brushing your hair), video capturing the modifications used to perform these tasks, and being inclusive of individuals with an LGMD at various stages of disease progression and mobility. In addition, as an outcome measure designed for use in a home setting, the LGVA intends to increase comfort and reduce travel burdens (both physical and financial) for participants. This initial development study for the LGVA will involve individuals with LGMD subtypes of 2A, 2B, and 2I.

The aims of the LGVA study are:

• To work together with members from the LGMDs 2A, 2B, and 2I communities to determine appropriate, everyday tasks that can be captured at home using a personal smart phone or other Wi-Fi-enabled device for an innovative functional assessment.
• To determine the reliability and sensitivity of the potential LGVA tasks.
• To collaborate with Key Opinion Leaders in the LGMDs community to analyze and determine the thresholds and scale used for assessing tasks and any potential functional ability changes.