Drug | Trial | Number of Patients | Comments* | References |
---|---|---|---|---|
Tazemetostat | NCT02601950 | 62 | “Efficacy was investigated in a single-arm cohort (Cohort 5) of a multi-center trial (Study EZH-202, NCT02601950) in patients with histologically confirmed, metastatic or locally advanced epithelioid sarcoma.” | |
Avapritinib | NCT02508532 | 43 | “Efficacy was investigated in NAVIGATOR (NCT02508532), a multi-center, single-arm, open-label trial enrolling 43 patients with GIST harboring a PDGFRA exon 18 mutation” | [10] |
Enfortumab Vedotin | NCT03219333 | 125 | “Efficacy was investigated in EV-201 (NCT03219333), a single-arm, multicenter trial enrolling 125 patients with locally advanced or metastatic urothelial cancer who received prior treatment with a PD-1 or PD-L1 inhibitor and platinum-based chemotherapy.” | [11] |
Zanubrutinib | NCT03206970 NCT02343120 | 86 32 | “Efficacy was evaluated in BGB-3111-206 (NCT03206970), a phase 2 open-label, multicenter, single-arm trial of 86 patients with MCL who received at least one prior therapy. Efficacy was also assessed in BGB-3111-AU-003 (NCT 02343120), a phase 1/2, open-label, dose-escalation, global, multicenter, single-arm trial of B‑cell malignancies, including 32 previously treated MCL patients treated with zanubrutinib administered orally at 160 mg twice daily or 320 mg once daily.” | [12] |
Entrectinib | ALKA NCT02097810 NCT02568267 | 54 51 | “Efficacy in NTRK-positive tumors was investigated in 54 adult patients who received entrectinib at various doses and schedules in one of three multicenter, single-arm, clinical trials: ALKA, STARTRK-1 (NCT02097810) and STARTRK-2 (NCT02568267)” “Efficacy in ROS1-positive metastatic NSCLC was investigated in 51 adult patients who received entrectinib at various doses and schedules in the same three trials; 90% received entrectinib 600 mg orally once daily.” | |
Selinexor | NCT02336815 | 122 | “Efficacy was evaluated in 122 patients enrolled in Part 2 of STORM (KCP-330-012; NCT02336815), a multicenter, single-arm, open-label study of patients with RRMM who had previously received three or more anti-myeloma treatment regimens including an alkylating agent, glucocorticoids, bortezomib, carfilzomib, lenalidomide, pomalidomide, and an anti-CD38 monoclonal antibody… …The approval was based on efficacy and safety in a prespecified subgroup analysis of 83 patients”. | [4] |