Table 3.
Other secondary endpoints.
|
Placebo (n = 27) |
Luvadaxistat 75 mg BID (n = 14) |
Luvadaxistat 300 mg BID (n = 26) |
|
|---|---|---|---|
| T25FW: change from baseline at week 12 (seconds) 1 | |||
| Patients, n | 11 | 5 | 10 |
| Mean (SD) | –0.29 (0.73) | 1.10 (2.57) | –1.25 (3.27) |
| Difference in LS mean (SE) vs placebo at week 12 | — | 2.18 (1.08) | 0.78 (0.82) |
| One‐sided p value vs placebo | — | 0.972 | 0.825 |
| FARS ADLs score: change from baseline at week 12 2 | |||
| Patients, n | 24 | 12 | 24 |
| Mean (SD) | –0.40 (2.62) | –0.29 (2.19) | –0.52 (2.02) |
| Difference in LS mean (SE) vs placebo at week 12 | – | 0.24 (0.82) | 0.37 (0.68) |
| One‐sided p value vs placebo | — | 0.616 | 0.708 |
| PGI‐S: change from baseline at week 12 3 | |||
| Patients, n | 24 | 12 | 24 |
| Patients, n (%) | |||
| Improved | 6 (25.0) | 5 (41.7) | 5 (20.8) |
| No change | 16 (66.7) | 6 (50.0) | 19 (79.2) |
| Worsened | 2 (8.3) | 1 (8.3) | 0 (0.0) |
| One‐sided p value vs placebo | — | 0.194 | 0.408 |
BID, twice daily; FARS ADLs, activities of daily living component of the Friedreich Ataxia Rating Scale; LS, least‐squares; PGI‐S, Patient Global Impression‐Severity scale; SD, standard deviation; SE, standard error; T25FW, timed 25‐foot walk.
1
T25FW was assessed only in participants who could walk. A negative change from baseline in T25FW indicates improvement.
2
A negative change from baseline in FARS ADLs score indicates improvement.
3
Change pertains to upper extremity functioning.