Table 1. Baseline Demographic and Clinical Characteristics (Full Analysis Set).
| Characteristic | Placebo (n = 64) | Methylcobalamin (n = 65) | Total (n = 129) |
|---|---|---|---|
| Male sex, No. (%) | 40 (63) | 34 (52) | 74 (57) |
| Age, mean (SD), y | 60.8 (12.1) | 61.2 (11.4) | 61.0 (11.7) |
| Period from ALS onset at the enrollment of the observation period, mean (SD), mo | 8.5 (2.3) | 8.2 (2.4) | 8.3 (2.3) |
| ALSFRS-R total score at baseline, mean (SD) | 42.3 (2.7) | 42.4 (2.6) | 42.4 (2.6) |
| FVC at baseline, mean (SD), % | 90.6 (16.9) | 93.4 (16.9) | 92.0 (16.9) |
| Body mass index, mean (SD) | 22.6 (3.9) | 21.8 (2.8) | 22.2 (3.4) |
| Vitamin B12 level at the enrollment of the observation period, mean (SD), pg/mL | 571.8 (719.9) | 585.9 (373.0) | 578.9 (570.2) |
| Disease type, No. (%) | |||
| Upper extremity | 32 (50) | 33 (51) | 65 (50) |
| Lower extremity | 13 (20) | 13 (20) | 26 (20) |
| Bulbar | 19 (30) | 19 (29) | 38 (30) |
| ALS type, No. (%) | |||
| Familial ALS | 0 (0) | 1 (2) | 1 (1) |
| Sporadic ALS | 64 (100) | 64 (98) | 128 (99) |
| Concomitant use of riluzole, No. (%) | 58 (91) | 58 (89) | 119 (92) |
| History of edaravone use, No. (%) | 6 (9) | 4 (6) | 10 (8) |
| ALS diagnosis of updated Awaji criteria, No. (%)a | |||
| Definite | 16 (25) | 23 (35) | 39 (30) |
| Probable | 32 (50) | 30 (46) | 62 (48) |
| Probable laboratory-supported | 16 (25) | 12 (19) | 28 (22) |
| ALS severity at baseline, No. (%)b | |||
| Grade 1 | 21 (33) | 21 (32) | 42 (33) |
| Grade 2 | 43 (67) | 44 (68) | 87 (67) |
| Change in ALSFRS-R total score in the observation period, No. (%)c | |||
| −2 | 28 (44) | 31 (48) | 59 (46) |
| −1 | 36 (56) | 34 (52) | 70 (54) |
Abbreviations: ALS, amyotrophic lateral sclerosis; ALSFRS-R, Revised Amyotrophic Lateral Sclerosis Functional Rating Scale; FVC, forced vital capacity.
The updated Awaji criteria, adopted as the ALS diagnostic criteria in this trial, comprised the categories of definite, probable, probable laboratory-supported, and possible. Patients with ALS who met the criteria of definite, probable, or probable laboratory-supported categories were eligible for enrollment.12
ALS severity: The severity of ALS symptoms was graded according to the Japan ALS severity classification of grades 1 to 5, with grade 5 being the most severe.
Change in the ALSFRS-R total score during the 12-week observation period before randomization. ALSFRS-R ranges from 0 to 48, with a lower score indicating more severe symptoms.