Table 2.
9‐HPT‐1 and mFARS‐neuro scores.
|
Placebo (n = 27) |
Luvadaxistat 75 mg BID (n = 14) |
Luvadaxistat 300 mg BID (n = 26) |
|
|---|---|---|---|
| Mean 9HPT at baseline | 66.37 (29.62) | 79.64 (42.93) | 66.43 (27.22) |
| 9‐HPT‐1 (1/seconds) | |||
| Baseline, mean (SD) | 0.01711 (0.005489) | 0.01571 (0.006871) | 0.01778 (0.007747) |
| Mean at week 12 (SD) 1 | 0.01798 (0.004780) | 0.01538 (0.005804) | 0.01750 (0.008345) |
| Mean change from baseline at week 12 (SD) | 0.00029 (0.003071) | –0.00031 (0.001033) | –0.00059 (0.001611) |
| Difference in LS mean (SE) vs placebo at week 12 | — | –0.00054 (0.000746) | –0.00069 (0.000616) |
| Adjusted one‐sided p value vs placebo | — | 1.00 | 1.00 |
| mFARS‐neuro total score | |||
| Baseline, mean (SD) | 50.83 (13.55) | 51.01 (13.64) | 51.12 (13.27) |
| Mean at week 12 (SD) 1 | 46.79 (12.75) | 49.27 (14.75) | 50.20 (13.69) |
| Mean change from baseline at week 12 (SD) | –2.95 (3.14) | –1.00 (3.37) | –1.43 (3.77) |
| Difference in LS mean (SE) vs placebo at week 12 | — | 2.02 (1.27) | 2.11 (1.05) |
| Adjusted one‐sided p value vs placebo | — | 0.942 | 0.975 |
A larger 9‐HPT‐1 score indicates better function; a negative change in 9‐HPT‐1 indicates worsened function. A negative change in mFARS‐neuro indicates improvement.
9‐HPT, nine‐hole peg test; 9‐HPT 1 , inverse of the time in seconds to complete the nine‐hole peg test; BID, twice daily; LS, least‐squares; mFARS‐neuro, modified Friedreich Ataxia Rating Scale neurological examination; SD, standard deviation; SE, standard error.
1
At week 12, n = 24 for placebo, n = 12 for luvadaxistat 75 mg BID, and n = 24 for luvadaxistat 300 mg BID.