Posted by Friends of FSH Research on Oct 14, 2020
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 have been able to maintain the work hours of our research coordinator Mory Mehrtash and devoted/devoting all the funding to support her salary.
At the University of Washington, we are currently running five trials in facioscapulohumeral dystrophy (FSHD): RESOLVE, ReDUX4, Wellstone extension study, Wellstone bilateral study, Oral Strength.
- ReSolve (Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD) study: We have enrolled 19 patients in this observational study as part of the FSHD Clinical Trial Research Network (CTRN). We trying to validate new clinical outcome assessments and refine trial planning strategies in FSHD. We are in the top four US recruiting sites, have followed up on our patients and added an additional patient to the national study, with plans for two more patients.
- ReDUX4: 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 one patient converting 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 six patients with three more follow-up appointments planned.
- Wellstone bilateral study: We have enrolled five out of 12 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 have added one more patient with one more planned. We are part of a three-site network involving University of Rochester and Kansas to recruit 36 patients.
- Oral strength study: We are starting the study.
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