Table 6 Similarities and differences among the three major Phase 1 trial experiences with advanced sarcoma patients on Phase 1 trials.
MDACC Phase 1 unit | Royal Marsden Hospital Phase 1 unit36 | European Phase 1 database37 | |
|---|---|---|---|
Number of patients | 100 | 133 | 178 |
Median no. of systemic therapies | 2 (0–10) | 3 (0–6) | 2 (0–9) |
Age range | 48 (14–80) | 48.0 (12.5–81.9) | 51 (19–79) |
Male/Female ratio | 46/54 | 71/62 | 96/82 |
Best Responses | 4 PR | 1 CR, 2PR. | 6 PR |
PFS | median PFS 3.5 months (95% CI: 2.4–4.7) 3 month and 6 month PFS, 57% and 36% | Median PFS 2.1 months (95% CI, 1.7–2.5) | 3-month and 6-month PFS rates were 33.5% and 16.9% |
Median OS | 9.6 months (95% CI: 8.1–14.2) | 7.6 months (95% CI, 4.8–10.4). | 9.8 months (39.4 weeks) (95% CI 27.4–51.4) |
Major tumor types | Spindle cell Sarcoma 12 (12%), Leiomyosarcoma 10 (10%) | Leiomyosarcoma 16 (12%), GIST 15 (11.3%), Liposarcoma 15 (11.3%) | Leiomyosarcoma 34 (27.2%), Liposarcoma 16 (12.8%) |
Major drug types | anti-VEGF–based (n = 45), mTOR inhibitor–based (n = 15), and anti-VEGF + mTOR inhibitor–based (n = 17) | Antiangiogenic 43 (32.3%), IGF1-R/PI3K/mTOR/AKT pathway 30 (22.6%) | Cytotoxic 43 (19.8%), Targeted or cytotoxic+ targeted 174 (80.2%) |
