Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study

Table 1 Schedule of assessments

Visits	Visit 1		Visit 2	Visit 3	Visit 4
Visits	Day 1	Day 2	Visit 2	Visit 3	Visit 4
Time (Month)	0		3	12	18
Time (Month)	0		+/− 1 Week	+/− 2 Weeks	+/− 2 Weeks
Obtain Consent	X
Confirm Eligibility	X
Urine Pregnancy Test^a	X		X	X	X
History and Physical Exam	X		X	X	X
Blood for FSHD DNA Testing	X		X
Blood for Serum Extraction	X		X	X	X
Blood for RNA (PAX gene tube)	X		X
Blood for Plasma (EDTA tube)	X		X	X	X
PROs: FSHD-HI, PROMIS57, UEFI, and FDI physical score	X		X		X
Electrical Impedance Myography	X	X	X	X	X
FSHD-COM / MFM Domain 1	X	X	X	X	X
Strength Testing: QMT and MMT	X		X	X	X
Bedside spirometry	X		X	X	X
DEXA	X			X
Clinical Severity Scores	X		X	X	X
Iowa Oral Performance Instrument	X	X	X	X	X
Reachable Workspace	X	X	X	X	X
Fall/Exercise Questionnaire	X		X	X	X
Fall Assessment^b			X
“Domain-delta” questionnaire			X	X

^aUrine pregnancy test for women of childbearing age, ^b weekly × 12 weeks after the month 3 visit
Assessments occur at baseline, 3, 12, and 18 months. The FSHD-COM, electrical impedance myography, MFM Domain 1, Iowa Oral Performance Instrument, and Reachable Workspace are performed on Day 1 and Day 2 of the baseline visit in order to establish intra-rater reliability

ISSN: 1471-2377