Design characteristics and assumptions of placebo-controlled trials in ALS with mortality endpoints
| Design characteristics | Assumed survival | Observed survival | ||||||
| Treatment ( year ) | No. patients | Event | Follow-up |
Treatment effect
(HR) |
Survival (%)
(at month) | Hazard rate | Survival (%) | Hazard rate |
| Creatine (2003) | 175 | D, T, N | 16 | 0.44 | 60 (16) | 3.2 | 53.5 | 3.9 |
| Xaliproden I (2004) | 867 | D, T, N | 18 | 0.62 | 50 (18) | 3.9 | 53.8 | 3.4 |
| Xaliproden II (2004) | 1210 | D, T, N | 18 | 0.66 | 57 (18) | 3.1 | 62.3 | 2.6 |
| Pentoxifylline (2006) | 400 | D | 18 | 0.65 | 40 (18) | 5.1 | 59.7 | 2.9 |
| Valproic acid (2009) | 163 | D, T, N | 16 | 0.56 | 60 (16) | 3.2 | 75.1 | 1.8 |
| Lithium (2012) | 133 | D, T, N | 16 | 0.56 | 60 (16) | 3.2 | 66.5 | 2.5 |
| Pioglitazone (2012) | 219 | D | ≥18 | 0.49 | 60 (18) | 2.8 | 69.6 | 2.0 |
| Dexpramipexole (2013) | 942 | D, T, N | 12–18 | 0.63 | 80 (12) | 1.9 | 80.7 | 1.8 |
| Lithium (2013) | 214 | D | 18 | 0.45 | 65 (18) | 2.4 | 59.4 | 2.9 |
| Ceftriaxone (2014) | 513 | D, T, N | ≥12 | 0.66 | 75 (12) | 2.4 | 74.6 | 2.4 |
| Olesoxime (2014) | 512 | D, T, N | 18 | 0.63 | 62 (18) | 2.7 | 67.2 | 2.2 |
| Erythropoietin (2015) | 208 | D, T, N | 12 | 0.33 | 58 (12) | 4.5 | 74.1 | 2.5 |
| Ozanezumab (2017) | 303 | D | 11 | 0.49 | 90 (11) | 0.9 | 96.1 | 0.4 |
Follow-up time is given in months. Number of patients is for the total sample size. Hazard rates are given in number of events per 100 person-months.
ALS, amyotrophic lateral sclerosis; D, death;N, non-invasive ventilation; T, tracheostomy.