Table 1.
Baseline demographic and clinical characteristics
| Characteristic | Dose escalation tablet (n = 18) | Dose escalation suspension (n = 26) | Dose expansion (n = 12) | Overall population (n = 56) |
|---|---|---|---|---|
| Median age, years (range) | 9.5 (6–17) | 9.0 (3–17) | 5.0 (3–29) | 9.0 (3–29) |
| Age group, n (%) | ||||
| 2 to < 6 years | 0 | 3 (12) | 7 (58) | 10 (18) |
| 6 to < 12 | 12 (67) | 17 (65) | 2 (17) | 31 (55) |
| 12 to < 18 | 6 (33) | 6 (23) | 2 (17) | 14 (25) |
| ≥ 18 | 0 | 0 | 1 (8) | 1 (2) |
| Sex, n (%) | ||||
| Male | 11 (61) | 17 (65) | 5 (42) | 33 (59) |
| Female | 7 (39) | 9 (35) | 7 (58) | 23 (41) |
| Lansky/Karnofsky score, n (%) | (n = 17) | (n = 21) | (n = 9) | (n = 47) |
| ≥ 90 | 12 (71) | 19 (90) | 8 (89) | 39 (83) |
| 50−80 | 5 (29) | 2 (10) | 1 (11) | 8 (17) |
| Tumor type, n (%) | ||||
| LGG | 7 (39) | 13 (50) | 12 (100) | 32 (57) |
| PN in NF1 | 4 (22) | 8 (31) | 0 | 12 (21) |
| HGG | 4 (22) | 1 (4) | 0 | 5 (9) |
| MPNST | 0 | 2 (8) | 0 | 2 (4) |
| Mediastinal yolk sac tumor | 1 (6) | 0 | 0 | 1 (2) |
| Neuroblastoma | 1 (6) | 0 | 0 | 1 (2) |
| Liposarcoma | 1 (6) | 0 | 0 | 1 (2) |
| ATRT | 0 | 1 (4) | 0 | 1 (2) |
| DNET in Noonan syndrome | 0 | 1 (4) | 0 | 1 (2) |
| Median weight, kg (range) |
36.3 (20.0–56.7) |
36.1 (18.1–98.1) |
23.1 (10.2–100.9) |
33.5 (10.2–100.9) |
| Median lines of prior therapya, n (range) | 5.0 (1–13) | 5.0 (0–18) | 4.5 (1–7) | 5.0 (0–18) |
| Prior radiotherapy, n (%) | 7 (39) | 7 (27) | 1 (8) | 15 (27) |
| Prior surgery, n (%) | 17 (94) | 20 (77) | 11 (92) | 48 (86) |
ATRT atypical teratoid/rhabdoid tumor, DNET dysembryoplastic neuroepithelial tumor, HGG high-grade glioma, LGG low-grade glioma, MPNST malignant peripheral nerve sheath tumor, PN in NF1 plexiform neurofibroma in neurofibromatosis type 1
aPrior therapy includes systemic therapy, radiotherapy, surgery, and stem cell transplant