Table 1.
Patient characteristics: PC-PG and other neuroendocrine tumors in phase II study of ONC201.
| Patient characteristic | PC-PG | Other neuroendocrine tumors |
|---|---|---|
| Patients with metastases (%) | 14 (100%) | 16 (100%) |
| Age, years, median (range) | 57 (18–70) | 35.5 (19–65) |
| Gender, N (%) | ||
| Male | 3 | 4 |
| Female | 11 | 12 |
| KPS score, median (range) | 90 (70–100) | 90 (80–100) |
| Weight, kg, median (range) | 91.2 (72.9–117.9) | 81.35 (60.7–108.9) |
| Height, cm, median (range) | 178 (159.3–198.3) | 180.7 (160–188) |
| Time from initial diagnosis, N (%) | ||
| <6 months | 0 (0) | 1 (6.3%) |
| >6 months to <2 years | 2 (14.3%) | 1 (6.3%) |
| >2 years to <5 years | 4 (28.6%) | 8 (50%) |
| >5 years to <10 years | 2 (14.3%) | 5 (31.3%) |
| >10 years to <20 years | 5 (35.7%) | 1 (6.3%) |
| >20 years | 1 (7.1%) | 0 (0) |
| Type of neuroendocrine cancer | ||
| PC-PG | 14 (100%) | |
| DSRCT | 10 (62.5%) | |
| MTC | 1 | 2 (12.6%) |
| Cholangiocarcinoma | 1 (6.3%) | |
| ACC | 1 (6.3%) | |
| Clear cell sarcoma | 1 (6.3%) | |
| Neuroblastoma | 1 (6.3%) | |
| Prior local treatments, N | ||
| Surgery only | 2 (14.3%) | 1 (6.3%) |
| Surgery + radiotherapy (RT) | 3 (21.4%) | 2 (12.5%) |
| Surgery + chemotherapy | 3 (14.3%) | 0 (0) |
| RT + chemotherapy | 1 (7.1%) | 0 (0) |
| Surgery + RT + chemotherapy | 5 (35.7%) | 13 (81.3%) |
| Sites of metastasis, N (%) | ||
| Lymph nodes | 11 (78.6%) | 12 (75%) |
| Lung | 6 (42.9%) | 13 (81.3%) |
| Liver | 2 (14.3%) | 8 (50%) |
| Bone | 13 (92.9%) | 7 (43.8%) |
| Other | 0 | 2 (12.5%) |
| Baseline plasma chromogranin, picogram/mL, median (range) | 697 (71–71,760) | N/A |
| Partial response: % change | −25, −4, −10, +6, +71% | |
| Stable disease: % change | −60,−36, +2, +7, +1, +73, +313% | |
| Progression: % change | +93% | |
| (1 patient not done) | ||
| Baseline plasma | ||
| Normetanephrine (median) | 284 (65–10,244) | |
| Partial response: % change | −66, −29, −19, −16, −8% | |
| Stable disease: % change | −60, −47, −25, −4, −3, 0, +20% | |
| Progression: % change | +113% | |
| (1 patient not done) | ||
| Germline mutations (N) | SDHA (2), SDHB (7) | RET (1) |
| SDHC (1), SDHD (1) | ||
| RET (1)a | ||
| Fumarate hydrase (1) | ||
| Somatic mutations (N) | NF1 p.F1778 fs | EWS-WT1(10), EWS-ATF (1), |
| ATRX fs +ZFHX3 fs (1) | ATRX p. V1557fs +ALKp.R1275 (1) | |
| GNAS R201C, KRAS G13D+ | ||
| PIK3 E542K (1) | ||
aAlso with PC-PG (MEN2).