Table 2.
Schedule of events
Visits | Baseline day 1 | Baseline day 2 | Month 3a ± 21 days | Month 6b
± 21 days |
Month 12 ± 21 days |
---|---|---|---|---|---|
Informed consent | X | ||||
Demographic information | X | ||||
Medical and surgical history | X | ||||
Inclusion exclusion | X | ||||
Vital signs | X | X | X | X | X |
Physical exam | Xc | Xc | X | X | X |
Pregnancy testd | X | X | X | X | |
10 Meter walk | X | ||||
100 m timed | X | X | X | X | X |
NSAD | X | X | X | X | X |
4 stair climb | X | X | X | X | X |
Timed Up and Go (TUG) | X | X | X | X | X |
Handheld dynamometry | X | X | X | X | X |
Performance Upper Limb PUL 2.0 | X | X | X | X | X |
9 hole peg test | X | X | X | X | X |
Spirometry | X | X | X | X | X |
Concomitant meds | X | X | X | X | X |
Domain delta questionnaire | X | X | X | X | |
Patient reported outcome measures | Xc | Xc | X | X | X |
Urine biomarkers | X | X | X | ||
Blood biomarkers | X | X | X | X | |
Prior muscle biopsy acquired (if performed) | X | ||||
Review adverse events | X | X | X | X |
aFor individuals with SGCA-G pathogenic variants
bn/a to DNAJB6/ LGMDD1cohort
cAssessment can occur on baseline day 1 or baseline day 2
dPregnancy test for women of childbearing potential