Table 2.
Primary and selected key secondary efficacy endpoints according to treatment randomization (intention‐to‐treat analysis)
| T2D | T1D | |||||
|---|---|---|---|---|---|---|
| Alirocumab (n = 287) |
Placebo (n = 142) |
Difference vs placebo, % [95% CI], P |
Alirocumab (n = 49) |
Placebo (n = 25) |
Difference vs placebo, % [95% CI], P |
|
| Mean (s.d.) calculated LDL cholesterol | ||||||
| Baseline | ||||||
| mmol/L | 2.9 (0.9) | 2.8 (1.0) | 3.2 (1.2) | 2.9 (0.8) | ||
| mg/dL | 110.3 (35.9) | 109.5 (38.7) | 122.5 (47.8) | 110.2 (31.2) | ||
| Week 24 | ||||||
| mmol/L | 1.4 (0.9) | 2.8 (1.0) | 1.4 (0.9) | 2.6 (0.8) | ||
| mg/dL | 55.1 (33.7) | 107.3 (37.0) | 55.9 (33.5) | 101.8 (31.8) | ||
| LS mean (s.e.) primary endpoint: % change from baseline to week 24 | −48.2 (1.6) | 0.8 (2.2) | −49.0 (2.7)[−54.4 to −43.6], <.0001 | −51.8 (3.7) | −3.9 (5.3) | −47.8 (6.5)[−60.7 to −35.0], <.0001 |
| Selected key secondary efficacy endpoints | ||||||
| % change from baseline, mean (s.e.) | ||||||
| Calculated LDL cholesterol (week 12) | −48.8 (1.4) | 1.4 (2.1) | −50.2 (2.5)[−55.2 to −45.3], <.0001 | −49.4 (3.5) | −4.5 (5.0) | −44.8 (6.1)[−56.9 to −32.8], <.0001 |
| Non‐HDL cholesterol (week 24) | −37.9 (1.4) | 0.7 (2.0) | −38.7 (2.4)[−43.4 to −33.9], <.0001 | −45.9 (3.3) | −3.2 (4.8) | −42.7 (5.8)[−54.2 to −31.3], <.0001 |
| ApoB (week 24) | −33.4 (1.3) | 3.3 (1.7) | −36.7 (2.1)[−40.9 to −32.5], <.0001 | −39.4 (3.0) | −0.4 (4.3) | −39.0 (5.3)[−49.4 to −28.7], <.0001 |
| Total cholesterol (week 24) | −26.8 (1.0) | 0.8 (1.5) | −27.6 (1.8)[−31.2 to −24.1], <.0001 | −29.9 (2.5) | −0.7 (3.6) | −29.2 (4.3)[−37.8 to −20.7], <.0001 |
| Lp(a) (week 24) | −19.0 (1.6) | −0.5 (2.2) | −18.4 (2.7)[−23.7 to −13.2], <.0001 | −23.0 (3.8) | −4.3 (5.3) | −18.7 (6.5)[−31.4 to −6.0], = .0039 |
| HDL cholesterol (week 24)a | 8.1 (1.0) | 3.7 (1.4) | 4.4 (1.7)[1.1 to 7.7], <.01 | 11.2 (2.4) | 7.3 (3.5) | 3.9 (4.1)[−4.2 to 12.0], = .3434b |
| Triglycerides (week 24)a | −5.7 (2.0) | 0.0 (2.7) | −5.7 (3.4)[−12.3 to 0.9], = .0902b | −13.6 (4.7) | 1.9 (6.7) | −15.5 (8.1)[−31.4 to 0.4], = .056b |
| LDL particle numbers (week 24) | −38.3 (1.3) | 1.9 (1.9) | −40.2 (2.3)[−44.7 to −35.6], <.0001b | −44.4 (3.2) | −4.4 (4.6) | −40.0 (5.6)[−51.0 to −28.9], <.0001b |
| LDL particle size (week 24) | −2.8 (0.1) | −0.3 (0.2) | −2.5 (0.2)[−2.9 to −2.0], <.0001b | −2.3 (0.3) | 0.8 (0.5) | −3.0 (0.6)[−4.2 to −1.9], <.0001b |
Abbreviations: CI, confidence interval; ITT, intention‐to‐treat.
a
Hierarchical testing terminated at the endpoint triglycerides in participants with T2D and at the endpoint HDL cholesterol in participants with T1D, therefore all subsequent statistical comparisons were not considered statistically significant.
b
P values for descriptive purposes only.