Table 2.
Summary of End Points Assessed by Blinded Independent Central Review.*
| End Point | Intention-to-Treat–Pembrolizumab Population | Overall Intention-to-Treat Population | ||
|---|---|---|---|---|
| Selpercatinib (N = 129) | Control (N = 83) | Selpercatinib (N = 159) | Control (N=102) | |
| Progression-free survival — mo | ||||
| Median progression-free survival (95% CI) | 24.8 (16.9–NE) | 11.2 (8.8–16.8) | 24.8 (17.3–NE) | 11.2 (8.8–16.8) |
| Median duration of follow-up (95% CI) | 19.4 (16.7–19.7) | 18.9 (14.2–22.3) | 19.4 (16.7–19.6) | 16.5 (13.6–21.0) |
| Objective response (95% CI) — % of patients | 84 (76–90) | 65 (54–75) | 84 (77–89) | 63 (53–72) |
| Best overall response — no. (%) | ||||
| Complete response | 9 (7) | 5 (6) | 12 (8) | 5 (5) |
| Partial response | 99 (77) | 49 (59) | 121 (76) | 59 (58) |
| Stable disease | 14 (11) | 20 (24) | 17 (11) | 26 (25) |
| Progressive disease | 2 (2) | 5 (6) | 2 (1) | 7 (7) |
| Not evaluable | 5 (4) | 4 (5) | 7 (4) | 5 (5) |
| Duration of response | ||||
| Patients with a response — no. | 108 | 54 | 133 | 64 |
| Patients with a response and censored data — no. (%) | 74 (69) | 25 (46) | 43 (32) | 31 (48) |
| Median duration of response (95% CI) — mo | 24.2 (17.9–NE) | 11.5 (9.7–23.3) | 24.2 (17.9–NE) | 12.0 (9.7–23.3) |
| Median duration of follow-up (95% CI) — mo | 18.0 (16.5–19.5) | 14.6 (11.2–19.8) | 17.9 (15.7–18.7) | 12.7 (11.1–16.6) |
*
Percentages may not total 100 because of rounding. Confidence intervals have not been adjusted for multiplicity and cannot be used to infer treatment effects. Efficacy outcomes were assessed with the use of Response Evaluation Criteria in Solid Tumors (RECIST), version 1.1, and were confirmed by blinded independent radiologic review. NE denotes not estimable.