Progress Update: Clinical Trial Support at University of Washington

Update by Dr. Wang
See grant  FlexFund: Clinical Trial Support at University of Washington

We would like to thank the Friends of FSH research for the FLEX Fund Grant to help support for our clinical research apparatus, due to the unexpected burden of the Fulcrum ReDUX4 study and the shuttering of operations due to COVID. We had been able to maintain the work hours of our research coordinator Mory Mehrtash.

At the University of Washington, we are currently running four studies in facioscapulohumeral dystrophy (FSHD): RESOLVE, ReDUX4 open-label extension, Wellstone extension study, Wellstone bilateral study.

  • ReSolve (Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD) study: We added more patients during the pandemic, and now have enrolled a total of 21 patients in this observational study as part of the FSHD Clinical Trial Research Network (CTRN). We continue to follow the patients longitudinally. We trying to validate new clinical outcome assessments and refine trial planning strategies in FSHD.
  • ReDUX4 Open-label extension: We have eight patients in the Fulcrum phase 2b trial of losmapimod. We are the second most active recruitment site. We have had multiple oversight visits from the clinical research organization each month, and have followed up on our eight patients; with all patients converted to the open-label phase.
  • Wellstone extension study: We enrolled 18 patients in the first Wellstone study resulting in three papers describing the longitudinal changes of the MRI, pathological, and clinical outcomes over one year in FSHD patients. Our study has informed the outcome measures the Fulcrum phase 2b trial and suggests that MRI can be used as a non-invasive and objective biomarker of ambulation in patients. We are currently adding a third visit four years after the first visit with muscle biopsy, MRI, and outcome measures. We have followed up on ten patients and hoping to re-engage five more patients for follow-up after the pandemic.
  • Wellstone bilateral study: We have completed enrollment of 11 patients in this study where we are validating our mRNA biomarkers for disease activity as well as single cell sequencing of the T-cell repertoire. We are part of a three-site network involving University of Rochester and Kansas.