Table 1.
Secondary efficacy outcomes from the phase 3 CHAPTER-GIST-301 trial of pimitespib in Japanese patients with gastrointestinal stromal tumours [71]
| Pimitespib (N = 58) | Placebo (N = 28) | P value | |
|---|---|---|---|
| Investigator-assessed PFS, median | |||
| Months (95% CI) | 2.9 (2.8–4.2) | 1.4 (0.8–2.5) | |
| HR (95% CI) | 0.58 (0.33–1.02) | – | 0.028a |
| Secondary PFSb, median | |||
| Months (95% CI) | 2.7 (0.7–1.4) | NA | |
| Adjusted OS, medianc | |||
| Months (95% CI) | 13.8 (9.2–NR) | 7.6 (5.3–14.9) | |
| HR (95% CI) | 0.42 (0.21–0.85) | – | 0.007a |
| Unadjusted OS, median | |||
| Months (95% CI) | 13.8 (9.2–NR) | 9.6 (5.5–NR) | |
| HR (95% CI) | 0.63 (0.32–1.12) | – | 0.081a |
| BCRR-assessed responses | |||
| Complete response, n (%) | 0 | 0 | |
| Partial response, n (%) | 0 | 0 | |
| Stable disease, n (%) | 36 (62.1) | 10 (35.7) | |
| Progressive disease, n (%) | 20 (34.5) | 18 (64.3) | |
| Not assessable, n (%) | 2 (3.4) | 0 | |
| DCR w/ duration ≥ 12 wk, % (95% CI) | 27.6 (16.7–40.9) | 21.4 (8.3–41) | 0.369d |
| SD w/ duration ≥ 6 wk, % (95% CI) | 62.1 (48.4–74.5) | 35.7 (18.6–55.9) | 0.019d |
| Time to progression, median | |||
| Months (95% CI) | 2.8 (1.6–2.9) | 1.4 (0.9–1.8) | |
| HR (95% CI) | 0.67 (0.41–1.09) | – | 0.052a |
BCRR blinded central radiological review, CI confidence interval, DCR disease control rate, HR hazard ratio, NA not applicable, NR not reached, OS overall survival, PFS progression-free survival, SD stable disease, w/ with, wk weeks
aOne-sided P value
bPFS in patients (n = 17) crossed over to pimitespib in the open-label phase
cAdjusted using the rank-preserving structural failure time
dFisher’s exact test, one-sided