Progress Update: Motor Outcomes to Validate Evaluations in FSHD

Report by Dr. Statland
See also Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

The primary goal of our Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) study is to hasten therapeutic development for FSHD and improve care delivery by creating a standard protocol informed by best practice guidelines to determine the predictive value of clinical characteristics and motor assessments on motor and health outcomes (i.e. need for orthotics, ambulation aides, respiratory support, etc.). The purpose of this study is to understand functional motor assessments, define which assessments are meaningful to people with FSHD, and if certain values of outcome assessments help predict future motor or health outcomes. MOVE FSHD is also unique in that it is truly meant to include all individuals with FSHD. Including those with FSHD type 2, children, and those who are in a wheelchair.

In our current study, despite an ongoing international pandemic, we have made considerable progress over the first 18 months.  One of the most difficult parts of running a multicenter study is performing the ethical review, contracting, standardizing the documents and forms across all institutions, and training study personnel.  We have completed training of 12 US academic institutions, including coordinators, evaluators, and principal investigators.  All 12 sites have obtained ethical approval, completed contracting, and are activated and enrolling. We are pleased to announce we have enrolled 140 participants, and plan to have all our planned 240 participants enrolled by the end of 2022.  This large study would be a big achievement in the FSHD community, a reality that has in a large part made this possible is the support from Friends of FSH Research.  Additionally, the MOVE FSHD study has also attracted the notice of industry and other funders – and we were able to add the MOVE+ sub-study that includes whole-body MRI and additional biomarkers, which are essential to accelerate future FSHD drug development.