Table 1.
Baseline Characteristics | Main cohort (n = 30) | Expanded access arm (n = 5) |
---|---|---|
% (n)/Mean (SD) | % (n)/Mean (SD) | |
Age at Screening (years) | 57.1 (10.3) | 53.8 (13.9) |
Family History of ALS | 10.0% (3) | 0.0% (0) |
Male | 53.3% (16) | 80.0% (4) |
Caucasian | 93.3% (28) | 100.0% (5) |
Symptom onset to Diagnosis (in months) | 13.2 (12.6) | 8.9 (6.4) |
Symptom onset to Screening (in months) | 24.4 (15.7) | 22.8 (9.0) |
Limb Onset | 66.7% (20) | 60.0% (3) |
Baseline SVC (% predicted) | 77.0 (23.2) | 81.0 (17.6) |
ALSFRS-R at Baseline | 37.2 (4.7) | 31.6 (11.6) |
Estimated ALSFRS-R Slope Pre-Baseline (48-Baseline ALSFRS-R/disease duration) (points/month) | 0.58 (0.37) | 0.70 (0.33) |
UMNB Total at Screening | 29.4 (3.5) | 27.0 (10.5) |
Exposure to 30-day stable dose of Riluzole | 83% (25) | 100% (5) |
Exposure to edaravone | 37% (11) | 20% (1) |