Table 3

Clinical efficacy (ORR per RECIST 1.1) and volumetric analysis

Dose levelPatient no.Pain improvementRECIST at the last assessment (3 months after first dose)Volumetric change, % change
(baseline/study end; cm3)
12NoPD (clin. deterioration)Not done
3NoPD (clin. deterioration)Not done
4NoPD (clin. deterioration)Not done
25NoPD+100 (15.3/30)
6NoPD+63.5 (7.4/12.1)
7YesSD+230 (57/188)
8NoSDNot done (too small to measure)
39YesPR−66 (2146/724)
10NoPD+75 (0.8/1.4)
11NoPD+212 (10.7/33.4)
12YesSD−41 (129/76)
13YesSD+27 (15.5/19.7)
14NoPD (SD per RECIST but with clin. deterioration)+25 (11.1/13.9)
All dose levelsn=13Yes 4 out of 13
No 9 out of 13
1 PR
4 SD
8 PD
Two with volumetric shrinkage
  • Summary of clinical efficacy results in all patients and per dose level, with a comparison of ORR per RECIST 1.1 and retrospective exploratory volumetric analysis.

  • ORR, objective response rate; PD, progressive disease; PR, partial response; RECIST, Response Evaluation Criteria in Solid Tumors; SD; stable disease