Table 1.
Demographic and baseline characteristics
| ATC (n = 36) | BTC (n = 43) | GIST (n = 1) | LGG (n = 13) | HGG (n = 45) | ASI (n = 3) | HCL (n = 55) | MM (n = 10) | Total (n = 206) | |
|---|---|---|---|---|---|---|---|---|---|
| Age (years) | |||||||||
| Mean (s.d.) | 69.6 (9.53) | 57.0 (11.88) | 77.0 () | 33.1 (11.51) | 41.9 (14.70) | 58.3 (3.21) | 64.8 (10.77) | 66.9 (6.89) | 57.1 (16.40) |
| Age group (years) | |||||||||
| <18 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| 18–64 | 9 (25%) | 29 (67%) | 0 | 13 (100%) | 43 (96%) | 3 (100%) | 21 (38%) | 4 (40%) | 122 (59%) |
| 65–74 | 13 (36%) | 13 (30%) | 0 | 0 | 2 (4%) | 0 | 24 (44%) | 5 (50%) | 57 (28%) |
| 75–84 | 12 (33%) | 1 (2%) | 1 (100%) | 0 | 0 | 0 | 9 (16%) | 1 (10%) | 24 (12%) |
| ≥85 | 2 (6%) | 0 | 0 | 0 | 0 | 0 | 1 (2%) | 0 | 3 (1%) |
| Sex | |||||||||
| Male | 16 (44%) | 19 (44%) | 0 | 4 (31%) | 23 (51%) | 2 (67%) | 47 (85%) | 5 (50%) | 116 (56%) |
| Female | 20 (56%) | 24 (56%) | 1 (100%) | 9 (69%) | 22 (49%) | 1 (33%) | 8 (15%) | 5 (50%) | 90 (44%) |
| Baseline ECOG | |||||||||
| 0 | 4 (11%) | 17 (40%) | 1 (100%) | 5 (38%) | 14 (31%) | 3 (100%) | 25 (45%) | 3 (30%) | 72 (35%) |
| 1 | 30 (83%) | 24 (56%) | 0 | 7 (54%) | 24 (53%) | 0 | 27 (49%) | 6 (60%) | 118 (57%) |
| 2 | 2 (6%) | 2 (5%) | 0 | 1 (8%) | 7 (16%) | 0 | 3 (5%) | 1 (10%) | 16 (8%) |
| Time since diagnosis (days) | 125.0 | 347.0 | 325.0 | 2536.0 | 525 | 595 | 4578 | 2359 | — |
| Median (range) | (14.0–4,606.0) | (26.0–3,224.0) | (−) | (45–9,367) | (59–9,549) | (147–1,014) | (88–12,126) | (1,107–5,740) | |
| Measurable disease at screening | 36 (100) | 43 (100) | 1 (100) | 13 (100) | 43 (96%) | 3 (100%) | — | — | — |
| Non-target lesions at screening | 29 (81) | 31 (72) | 0 | 3 (23) | 10 (22%) | 1 (33%) | — | — | — |
| Stage, n (%) | |||||||||
| I | 0 | 0 | 0 | 6 (46) | 0 | 0 | — | — | |
| II | 0 | 1 (2) | 0 | 7 (54) | 0 | 0 | — | — | |
| III | 0 | 0 | 0 | 0 | 13 (29%) | 0 | — | — | |
| IV | 1 (3) | 1 (2) | 1 (100) | 0 | 31 (69%) | 2 (67%) | — | — | — |
| IVA | 0 | 0 | 0 | — | — | 1 (33%) | — | — | — |
| IVB | 0 | 40 (93) | 0 | — | — | 0 | − | — | — |
| IVC | 35 (97) | 0 | 0 | — | — | 0 | — | — | — |
| Missing | 0 | 1 (2) | 0 | 0 | 1 (2%) | 0 | — | — | — |
| Prior radiotherapy regimens | |||||||||
| 0 | 7 (19) | 38 (88) | 1 (100) | 5 (38) | 1 (2%) | 3 (100%) | — | — | — |
| 1 | 18 (50) | 4 (9) | 0 | 7 (54) | 36 (80%) | 0 | — | — | — |
| 2 | 11 (31) | 1 (2) | 0 | 1 (8) | 7 (16%) | 0 | — | — | — |
| 3 | 0 | 0 | 0 | 0 | 1 (2%) | 0 | — | — | — |
| Prior anti-cancer therapy | |||||||||
| Any therapy | 36 (100) | 43 (100) | 1 (100) | 12 (92) | 45 (100) | 3 (100) | 55 (100) | 10 (100) | 205 (>99) |
| Biologic therapy | 0 | 5 (12) | 0 | 2 (15) | 7 (16) | 2 (67) | 45 (82) | 4 (40) | 65 (32) |
| Chemotherapy | 15 (42) | 42 (98) | 0 | 5 (38) | 42 (93) | 3 (100) | 55 (100) | 10 (100) | 172 (83) |
| Hormonal therapy | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Immunotherapy | 4 (11) | 2 (5) | 0 | 0 | 1 (2) | 0 | 15 (27) | 10 (100) | 32 (16) |
| Radioactive therapy | 11 (31) | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 11 (5) |
| Small molecule targeted therapy | 7 (19) | 3 (7) | 1 (100) | 0 | 3 (7) | 0 | 5 (9) | 10 (100) | 29 (14) |
| Radiotherapy | 30 (83) | 5 (12) | 0 | 8 (62) | 44 (98) | 0 | 1 (2) | 7 (70) | 95 (46) |
| Surgery | 30 (83) | 24 (56) | 1 (100) | 12 (92) | 42 (93) | 3 (100) | 6 (11) | 3 (30) | 121 (59) |
Population is the efficacy evaluable population.
Values are for primary and expansion cohorts combined for all cancers except disease characteristics for GIST, LGG and ASI, which include the primary analysis cohort.