Table 3.
Baseline characteristics for the overall (ITT) masitinib 4.5 mg/kg/day cohort versus associated placebo group (regardless of baseline ΔFS or individual ALSFRS-R component scores) and for the enriched cohort most closely matched to the AB19001 study design.
| M4.5, regardless of baseline ALSFRS-R or ΔFS | M4.5 (⩾2 each baseline ALSFRS-R item, ΔFS < 1.1) | |||||
|---|---|---|---|---|---|---|
| PBO (N = 133) | M4.5 (N = 130) | Deltab (%) | PBO (N = 62) | M4.5 (N = 45) | Deltab (%) | |
| Sex; n (%) | ||||||
| Male | 80 (60.2) | 83 (63.8) | +3.6 | 39 (62.9) | 31 (68.9) | −6.0 |
| ΔFS < 1.1; n (%) | ||||||
| Yes | 114 (85.7) | 106 (81.5) | −4.2 | 62 (100.0) | 45 (100.0) | 0 |
| Average ΔFS (points/month) | ||||||
| Mean ± SD | 0.71 ± 0.69 | 0.73 ± 0.63 | +2.8 | 0.41 ± 0.23 | 0.39 ± 0.25 | −4.9 |
| Range | 0.05; 5.00 | 0.03; 3.69 | 0.05; 1.07 | 0.03; 1.01 | ||
| Average ALSFRS-R score | ||||||
| Mean ± SD | 38.1 ± 5.5 | 37.5 ± 5.5 | −1.6 | 42.0 ± 3.2 | 42.2 ± 3.1 | +0.5 |
| Range | 21.0; 47.0 | 23.0; 47.0 | 34.0; 47.0 | 36.0; 47.0 | ||
| Age (years) | ||||||
| Mean ± SD | 55.2 ± 10.6 | 55.5 ± 10.6 | +0.5 | 55.0 ± 10.1 | 55.6 ± 11.5 | +1.1 |
| Range | 27.0; 75.0 | 24.0; 79.0 | 28.0; 73.0 | 24.0; 78.0 | ||
| ALS diagnosis; n (%) | ||||||
| Definite | 79 (59.4) | 76 (58.5) | +0.9 | 39 (62.9) | 25 (55.6) | −7.3 |
| Probable | 43 (32.3) | 44 (33.8) | −1.5 | 19 (30.6) | 16 (35.6) | +5.0 |
| Probable, lab | 11 (8.3) | 10 (7.7) | +0.6 | 4 (6.5) | 4 (8.9) | +2.4 |
| Disease durationa (months) | ||||||
| Mean ± SD | 9.0 ± 6.8 | 9.7 ± 8.4 | +7.8 | 7.3 ± 5.9 | 8.2 ± 7.6 | +12.3 |
| FVC (predicted) | ||||||
| Mean ± SD | 89.2 ± 18.7 | 87.5 ± 16.9 | +1.9 | 92.9 ± 17.9 | 93.8 ± 17.0 | +1.0 |
| Site of onset; n (%) | ||||||
| Spinal | 109 (82.0) | 107 (82.3) | +0.3 | 46 (74.2) | 35 (77.8) | +3.6 |
| Bulbar | 24 (18.0) | 23 (17.7) | −0.3 | 16 (25.8) | 10 (22.2) | −3.6 |
| Region; n (%) | ||||||
| North America and Western Europe | 86 (64.7) | 81 (62.3) | −2.4 | 35 (56.5) | 24 (53.3) | −3.2 |
| Eastern Europe | 8 (6.0) | 8 (6.2) | +0.2 | 4 (6.5) | 4 (8.9) | +2.4 |
| Other countries | 39 (29.3) | 41 (31.5) | +2.2 | 23 (37.1) | 17 (37.8) | +0.7 |
a
Average disease duration from time of diagnosis.
b
Delta, relative difference between treatment arms (%) with respect to placebo.
ALSFRS-R, Amyotrophic Lateral Sclerosis Functional Rating Scale-revised; FVC, forced vital capacity; M4.5, masitinib 4.5 mg/kg/day; PBO, placebo; SD, standard deviation; ΔFS, ALSFRS-R progression rate from disease onset to baseline.