First-in-human study of GFH018, a small molecule inhibitor of transforming growth factor-β receptor I inhibitor, in patients with advanced solid tumors

Table 4 Tumor response assessed by investigators (RECIST 1.1)

	GFH018 5 mg BID 14d-on/14d-off (N = 4) n (%)	GFH018 10 mg BID 14d-on/14d-off (N = 3) n (%)	GFH018 20 mg BID 14d-on/14d-off (N = 4) n (%)	GFH018 30 mg BID 14d-on/14d-off (N = 7) n (%)	GFH018 40 mg BID 14d-on/14d-off (N = 4) n (%)	GFH018 50 mg BID 14d-on/14d-off (N = 4) n (%)	GFH018 65 mg BID 14d-on/14d-off (N = 6) n (%)	GFH018 85 mg BID 7d-on/7d-off (N = 6) n (%)	GFH018 85 mg BID 14d-on/14d-off (N = 12) n (%)	Total (N = 50) n (%)
BOR
CR	0	0	0	0	0	0	0	0	0	0
PR	0	0	0	0	0	0	0	0	0	0
SD	0	1 (33.3%)	1 (25.0%)	1 (14.3%)	0	1 (25.0%)	1 (16.7%)	1 (16.7%)	3 (25.0%)	9 (18.0%)
PD	2 (50.0%)	2 (66.7%)	1 (25.0%)	3 (42.9%)	3 (75.0%)	1 (25.0%)	5 (83.3%)	2 (33.3%)	4 (33.3%)	23 (46.0%)
NE	0	0	1 (25.0%)^e	0	0	0	0	0	0	1 (2.0%)^e
Non-CR/Non-PD^a	0	0	0	0	0	0	0	0	0	0
NA^b	2 (50.0%)	0	1 (25.0%)	3 (42.9%)	1 (25.0%)	2 (50.0%)	0	3 (50.0%)	5 (41.7%)	17 (34.0%)
ORR (90%CI)^c	0	0	0	0	0	0	0	0	0	0
DCR (90%CI)^d	0	1 (33.3%) (0.84%, 90.57%)	1 (25.0%) (0.63%, 80.59%)	1 (14.3%) (0.36%, 57.87%)	0	1 (25.0%) (0.63%, 80.59%)	1 (16.7%) (0.42%, 64.12%)	1 (16.7%) (0.42%, 64.12%)	3 (25.0%) (5.49%, 57.19%)	9 (18.0%) (8.58%, 31.44%)

Data are shown as n (%) unless otherwise specified. Responses were assessed in accordance with Response Evaluation Criteria in Solid Tumors version 1.1
Abbreviations: BOR best overall response, CR complete response, DCR disease control rate, NE not estimable, NA not available, ORR objective response rate, PD progressive disease, PR partial response, SD stable disease
^aPersistence of 1 or more nontarget lesion(s) and/or maintenance of tumor marker level above the normal limits
^bNo postbaseline assessments after the start date
^cDefined as the proportion of patients with CR and PR
^dDefined as the proportion of patients with CR, PR, and SD
^eSD of insufficient duration (<6 weeks after the start date without further evaluable tumor assessment)

ISSN: 1471-2407