Table 2.
Representative Recent Phase ≥ II Clinical Trials for Recurrent GBM Patients Evaluating Non-angiogenic, Biologic-Based Therapeutics
| Agent | # Patients | Response Criteria |
# Recurrences | Primary trial endpoint | ORR (%) | mOS [Weeks] |
Citation |
|---|---|---|---|---|---|---|---|
| Rilotumumab | 61 | Macdonald | ≤3 | ORR | 0 | 23.5 | Wen et al50 |
| Cilengitide | 81 | Macdonald | ≤1 | PFS6 | 9 | 43 | Reardon et al57 |
| Enzastaurin | 174 | Levin | ≤2 | PFS | 2.9 | 28.7 | Wick et al39 |
| Erlotinib | 54 | Macdonald | ≤1 | PFS6 | 3.7 | 33.5 | van den Bent et al58 |
| Erlotinib | 48 | WHO | ≤1 | ORR | 6.3 | 42.1 | Yung et al59 |
| Erlotinib + sirolimus | 32 | Macdonald | 1 | PFS6 | 0 | 33.8 | Reardon et al51 |
| Fenretinib | 23 | Not stated | ≤2 | PFS6 | 0 | 30 | Puduvalli et al52 |
| Imatinib | 51 | Macdonald | ≤2 | ORR or PFS6 | 5.9 | 23 | Raymond et al60 |
| Imatinib + hydroxyurea | 33 | Macdonald | No limit | PFS6 | 9 | 48.9 | Reardon et al61 |
| Imatinib + hydroxyurea | 231 | Macdonald | 1 | ORR | 3.4 | 26 | Reardon et al62 |
| Imatinib + hydroxyurea | 120 | Macdonald | 1 | PFS | 1.7 | 21 | Dresemann et al34 |
| Temsirolimus | 65 | Macdonald | ≤2 | PFS6 | 0 | 19 | Galanis et al53 |
| Vorinostat | 66 | Macdonald | ≤2 | ORR | 3.0 | 24.8 | Galanis et al43 |
| Perifosine | 16 | RANO | No limit | PFS6 | 0 | 14.7 | Kaley et al54 |
| Buparlisib | 65 | RANO | ≤2 | PFS6 | 0 | 42.6 | Wen et al55 |
| Marizomib | 30 | RANO | ≤2 | ORR | 3.3 | 39.5 | Bota et al63 |
| Selinexor | 68 (24 50 mg 2xWK) (14, 60 mg 2xWK) (30, 80 mg 1xWK) |
RANO | ≤3 | PFS6 | 8.3 (50 mg) 7.7 (60 mg) 10 (80 mg) |
45.6 33.5 43.5 |
Lassman et al64 |
| † Dabrafenib + trametinib (in BRAF mutant rGBM)* | 31 | RANO | 1 | ORR | 32 | 59.5 | Wen et al56 |
†Believed to be a “positive” study, with ORR = 32% and mOS of more than a year.
*Note this involved an enriched patient population.
PFS = Progression-free survival.
PFS6 = Proportion of patients with PFS at or beyond 6 months from start of treatment (or randomization).
OS = Overall survival.