Table 2.
Visits | Baseline Day 1 | Baseline Day 2 | Month 31 +/− 21 days | Month 62 +/− 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 | X3 | X3 | X | X | X |
Pregnancy Test4 | X | X | X | X | |
10 Meter Walk | X | ||||
100 Meter 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 | X3 | X3 | 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 |
For individuals with SGCA-G pathogenic variants
n/a to DNAJB6/ LGMDD1cohort
Assessment can occur on baseline day 1 or baseline day 2.
Pregnancy test for women of childbearing potential