Table 3. Safety End Points in the Safety Population.
| Adverse event | No. (%) | |||
|---|---|---|---|---|
| Q2W cohort (n = 74) | Q4W cohort (n = 79) | |||
| Alirocumab (n = 49) | Placebo (n = 25) | Alirocumab (n = 52) | Placebo (n = 27) | |
| Any adverse eventa | 26.0 (53.1) | 13.0 (52.0) | 26.0 (50.0) | 16.0 (59.3) |
| Most common adverse eventsa | ||||
| Laryngitis | 0 | 0 | 1.0 (1.9) | 0 |
| Nasopharyngitis | 7.0 (14.3) | 2.0 (8.0) | 1.0 (1.9) | 2.0 (7.4) |
| Pharyngitis | 1.0 (2.0) | 0 | 0 | 1.0 (3.7) |
| Rhinitis | 1.0 (2.0) | 1.0 (4.0) | 1.0 (1.9) | 0 |
| Sinusitis | 1.0 (2.0) | 0 | 0 | 0 |
| Tonsillitis | 3.0 (6.1) | 1.0 (4.0) | 0 | 1.0 (3.7) |
| Upper respiratory tract infection | 3.0 (6.1) | 3.0 (12.0) | 3.0 (5.8) | 3.0 (11.1) |
| Headache | 3.0 (6.1) | 2.0 (8.0) | 4.0 (7.0) | 1.0 (3.7) |
| Migraine | 0 | 2.0 (8.0) | 0 | 0 |
| Adverse events of special interest | ||||
| Injection site reaction | 3.0 (6.1) | 0 | 2.0 (3.8) | 0 |
| General allergic reaction | 0 | 0 | 2.0 (3.8) | 0 |
| Disturbance in attention and memory | 0 | 0 | 1.0 (1.9) | 0 |
| Hypoesthesia | 0 | 0 | 1.0 (1.9) | 0 |
| Adverse events leading to discontinuation | ||||
| Disturbance in attention and memory | 0 | 0 | 1.0 (1.9) | 0 |
| Syncope | 0 | 0 | 1.0 (1.9) | 0 |
| Serious adverse event | ||||
| Appendicitis | 0 | 1.0 (4.0) | 0 | 0 |
| Depression | 1.0 (2.0) | 0 | 0 | 0 |
| Abdominal hernia | 1.0 (2.0) | 0 | 0 | 0 |
| Abdominal pain | 1.0 (2.0) | 0 | 0 | 0 |
| Sympathetic posterior cervical syndrome | 1.0 (2.0) | 0 | 0 | 0 |
| Syncope | 0 | 0 | 2.0 (3.8) | 0 |
| Noncardiac chest pain | 0 | 0 | 0 | 1.0 (3.7) |
| Treatment-related adverse event | 4.0 (8.2) | 1.0 (4.0) | 7.0 (13.5) | 0 |
| Developmental parameters | ||||
| Height change from baseline at week 24, mean (SD), cm | 2.73 (4.15) | 2.13 (2.28) | 1.68 (2.11) | 1.75 (1.27) |
| Weight change from baseline at week 24, mean (SD), kg | 1.71 (2.47) | 1.89 (2.91) | 1.66 (3.24) | 2.15 (2.86) |
| Cogstate Battery test change from baseline at week 24, mean (SD) | ||||
| Detection test | −0.05 (0.07) | −0.04 (0.13) | −0.04 (0.08) | −0.04 (0.06) |
| Identification test | −0.05 (0.07) | −0.04 (0.08) | −0.05 (0.08) | −0.02 (0.13) |
| One Card Learning test | 0.03 (0.12) | −0.02 (0.24) | −0.06 (0.11) | 0.04 (0.11) |
| Groton Maze Learning test | 4.04 (14.57) | −13.13 (29.83) | −0.31 (24.75) | 5.19 (16.37) |
| Composite score | −0.313 (0.444) | −0.403 (1.008) | −0.136 (0.637) | −0.218 (0.501) |
| Tanner stage, boys | ||||
| Prepubescent | ||||
| Baseline | 4 (21.1) | 1 (5.9) | 0 | 5 (41.7) |
| Week 24 | 3 (17.6) | 0 | 0 | 1 (9.1) |
| Pubescent | ||||
| Baseline | 13 (68.4) | 13 (76.5) | 14 (77.8) | 4 (33.3) |
| Week 24 | 11 (64.7) | 13 (76.5) | 12 (70.6) | 7 (63.6) |
| Postpubescent | ||||
| Baseline | 2 (10.5) | 3 (17.6) | 4 (22.2) | 3 (25.0) |
| Week 24 | 3 (17.6) | 4 (23.5) | 5 (29.4) | 3 (27.3) |
| Tanner stage, girls | ||||
| Prepubescent | ||||
| Baseline | 4 (13.3) | 1 (12.5) | 7 (20.6) | 1 (6.7) |
| Week 24 | 4 (13.3) | 1 (12.5) | 2 (7.4) | 1 (8.3) |
| Pubescent | ||||
| Baseline | 16 (53.3) | 6 (75.0) | 13 (38.2) | 8 (53.3) |
| Week 24 | 15 (53.6) | 5 (62.5) | 16 (59.3) | 6 (50.0) |
| Postpubescent | ||||
| Baseline | 10 (33.3) | 1 (12.5) | 14 (41.2) | 6 (40.0) |
| Week 24 | 9 (32.1) | 2 (25.0) | 9 (33.3) | 5 (41.7) |
| hs-CRP, mean (SD), mg/L | ||||
| Baseline | 2.4 (7.9) | 0.6 (0.8) | 1.7 (3.4) | 0.4 (0.3) |
| Week 24 | 1.1 (1.6) | 1.2 (2.1) | 1.8 (4.6) | 0.6 (0.6) |
Abbreviations: hs-CRP, high-sensitivity C-reactive protein; Q2W, dosing every 2 weeks; Q4W, dosing every 4 weeks.
a
Treatment-emergent adverse events with high-level term ≥5% in any treatment group in the safety population.