Table 1.
Demographic and Clinical Characteristics of the Participants at Baseline (Modified Intention-to-Treat Population).*
| Characteristic | Sodium Phenylbutyrate–Taurursodiol (N = 87) | Placebo (N = 48) | Overall (N = 135) |
|---|---|---|---|
| Male sex — no. (%) | 61 (70) | 32 (67) | 93 (69) |
| White race — no. (%)† | 82 (94) | 46 (96) | 128 (95) |
| Age — yr | 57.6±10.4 | 57.3±7.6 | 57.5±9.5 |
| Bulbar onset — no. (%) | 26 (30) | 10 (21) | 36 (27) |
| Riluzole or edaravone use — no. (%)‡ | 62 (71) | 42 (88) | 104 (77) |
| Riluzole | 59 (68) | 37 (77) | 96 (71) |
| Edaravone | 22 (25) | 24 (50) | 46 (34) |
| Both | 19 (22) | 19 (40) | 38 (28) |
| Prebaseline ALSFRS-R slope§ | 0.95±0.43 | 0.93±0.60 | 0.94±0.49 |
| Slow vital capacity — % of predicted normal value | 83.6±18.2 | 83.9±15.9 | 83.7±17.4 |
| ALSFRS-R total score§ | 35.7±5.8 | 36.7±5.1 | 36.0±5.5 |
| Bulbar score | 9.5±2.4 | 10.0±2.6 | 9.7±2.5 |
| Fine-motor score | 8.0±2.7 | 8.0±2.6 | 8.0±2.7 |
| Gross-motor score | 7.5±2.8 | 7.6±2.6 | 7.6±2.8 |
| Breathing score | 10.6±1.9 | 11.0±1.8 | 10.8±1.9 |
| ATLIS upper-limb score — % of predicted normal valued¶ | 54.8±24.4 | 51.4±25.2 | 53.6±24.6 |
| ATLIS lower-limb score — % of predicted normal valued¶ | 57.6±24.9 | 57.1±25.8 | 57.4±25.1 |
| ATLIS total score — % of predicted normal valued¶ | 56.8±20.1 | 53.9±20.9 | 55.8±20.4 |
| Months since ALS symptom onset | 13.5±3.8 | 13.6±3.6 | 13.5±3.8 |
| Months since ALS diagnosis | 5.9±3.3 | 6.3±3.2 | 6.0±3.3 |
| Body-mass index‖ | 26.9±4.4 | 26.4±5.8 | 26.7±4.9 |
Plus–minus values are means ±SD. ALS denotes amyotrophic lateral sclerosis.
Race was reported by the trial participant.
Data are for use at or before trial entry.
The Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ALSFRS-R) measures 12 items in four domains of function, each scored on a scale from 0 to 4, with higher scores indicating better function.21 The prebaseline ALSFRS-R slope was defined as the rate of decline in the total score from symptom onset to baseline.
Values for the Accurate Test of Limb Isometric Strength (ATLIS) were standardized to the percentage of the predicted normal value on the basis of sex, age, weight, and height.24
The body-mass index is the weight in kilograms divided by the square of the height in meters.