Table 5.
Patient characteristics in reported studies
| Study | Age at onset (years) | Age at diagnosis (years) | Female | Ethnicity (white) | GAA1 repeat length | Ambulatory status | Pes cavus status | mFARSa | History |
|---|---|---|---|---|---|---|---|---|---|
| Part 1 [10] |
15.3 ± 4.8 (PBOb) 14.8 ± 4.8 (Omavc) Mean 25.6 |
24.4 ± 6.7 (PBO) 25.9 ± 6.4 (Omav) |
52% (Omav) 59% (PBO) |
94% (PBO) 98% (Omav) |
863 ± 278 (PBO) 700 ± 277 (Omav) |
94% (PBO) 89% (Omav) |
59% (PBO) 42% (Omav) |
40.5 ± 10 (PBO) 41.3 ± 12 (Omav) |
Areflexia 77% (PBO) 81% (Omav) |
| Scoliosis surgery 18% (PBO) 12% (Omav) | |||||||||
| Part 2 [23] |
15.6 ± 5.3 (PBO) 13.87 ± 4.7 (Omav) |
24.1 ± 7.8 (PBO) 23.4 ± 6.1 (Omav) |
33% (Omav) 61% (PBO) |
96.2% (PBO) 98% (Omav) |
676.2 ± 267.9 (PBO) 736.8 ± 206.8 (Omav) |
94% (PBO) 90% (Omav) |
5.2% (PBO) 5.1% (Omav) |
37.8 ± 10.8 (PBO) 40.8 ± 10.2 (Omav) |
Cardiomyopathy 29% (PBO) 49% (Omav) |
| Scoliosis 71% (PBO) 77% (Omav) | |||||||||
| Scoliosis surgery 19% (PBO) 31% (Omav) | |||||||||
| Part 3 [24] |
15.1 ± 5.3 (PBO-Omav) 15.9 ± 5.7 (Omav-Omav) |
23.6 ± 7.8 (PBO) 24.2 ± 6.5 (Omav) |
33% (PBO-Omav) 60% (Omav-Omav) |
93% (PBO-Omav) 98% (Omav-Omav) |
693.8 ± 277.2 (PBO-Omav) 739.2 ± 214.9 (Omav-Omav) |
93% in both the groups | - |
38.8 ± 11 (PBO) 40.9 ± 10.4 (Omav) |
Cardiomyopathy 29% (PBO-Omav) 48% (Omav-Omav) |
Values are mean ± SD
amcodified Friedreich’s Ataxia Rating Scale
bPlacebo
cOmaveloxolone treated