Table 2:

Tumor response by RECIST and TVS at week 25

	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.