Table 2:
Pexidartinib n=61 |
Placebo n=59 |
Difference in % (pexidartinib – placebo)* |
|
---|---|---|---|
Response rate based on RECIST for part 1 (primary endpoint) | |||
Complete response | |||
no. (%) | 9 (15) | 0 | |
95% CI | 8 to 26 | 0 to 6 | |
Partial response | |||
no. (%) | 15 (25) | 0 | |
95% CI | 16 to 37 | 0 to 6 | |
Stable disease | |||
no. (%) | 24 (39) | 46 (78) | |
95% CI | 28 to 52 | 66 to 87 | |
Progressive disease | |||
no. (%) | 1 (2) | 1 (2) | |
95% CI | 0 to 9 | 0 to 9 | |
Not evaluable† | |||
no. (%) | 12 (20) | 12 (20) | |
95% CI | 12 to 31 | 12 to 32 | |
Overall response (complete or partial) | |||
no. (%) | 24 (39)‡ | 0 | 39 |
95% CI | 28 to 52 | 0 to 6 | 27 to 52 |
Fisher’s exact test p value (one-sided) | <0·0001 | ||
Response rate based on TVS for part 1 | |||
Complete response | |||
no. (%) | 3 (5) | 0 (0) | |
95% CI | 2 to 14 | 0 to 6 | |
Partial response | |||
no. (%) | 31 (51) | 0 (0) | |
95% CI | 39 to 63 | 0 to 6 | |
Stable disease | |||
no. (%) | 14 (23) | 45 (76) | |
95% CI | 14 to 35 | 64 to 85 | |
Progressive disease | |||
no. (%) | 1 (2) | 2 (3) | |
95% CI | 0 to 9 | 0 to 12 | |
Not evaluable† | |||
no. (%) | 12 (20) | 12 (20) | |
95% CI | 12 to 31 | 12 to 32 | |
Overall response (complete or partial) | |||
no. (%) | 34 (56)‡ | 0 (0) | 56 |
95% CI | 43 to 68 | 0 to 6 | 42 to 68 |
Fisher’s exact test p value (one-sided) | <0·0001 |
95% confidence interval was calculated using the Newcombe method.
19 patients discontinued the study prior to their end of part 1 assessment, 3 patients had their MRI completed outside of the defined week 25 window (days 155–183), and 2 patients started part 2 treatment prior to their end of part 1 assessment.
Response rate increased to 53% by RECIST and 64% by TVS at the January 31, 2018 data cutoff, when tumor assessments after week 25 were included.
CI=confidence interval. MRI=magnetic resonance imaging, RECIST=Response Evaluation Criteria for Adverse Events, version 1.1. TVS=tumor volume score.