Selpercatinib

A phase I/II study (LIBRETTO-001 [NCT03157128]) enrolled patients with RET fusion−positive solid tumors. RET fusion status was determined by local molecular testing (NGS, FISH, or polymerase chain reaction assay) without central confirmation. The primary end point was objective response.[84][Level of evidence C3]

A phase I/II study (LIBRETTO-001 [NCT03157128]) enrolled patients with RET fusion−positive solid tumors. RET fusion status was determined by local molecular testing (NGS, FISH, or polymerase chain reaction assay) without central confirmation. The primary end point was objective response.[84][Level of evidence C3]

Updated analysis was conducted in 316 patients with RET fusion–positive NSCLC.[84]

A phase I/II study (LIBRETTO-001 [NCT03157128]) enrolled patients with RET fusion−positive solid tumors. RET fusion status was determined by local molecular testing (NGS, FISH, or polymerase chain reaction assay) without central confirmation. The primary end point was objective response.[84][Level of evidence C3]

Among the 69 treatment-naïve patients, the objective response rate was 84% (95% CI, 73%–92%), and 6% achieved complete responses. The median DOR was 20.2 months (95% CI, 13.0–could not be evaluated); 40% of responses were ongoing at the data cutoff (median follow-up, 20.3 months). The median PFS was 22.0 months; 35% of patients were alive and progression-free at the data cutoff (median follow-up, 21.9 months).

A phase I/II study (LIBRETTO-001 [NCT03157128]) enrolled patients with RET fusion−positive solid tumors. RET fusion status was determined by local molecular testing (NGS, FISH, or polymerase chain reaction assay) without central confirmation. The primary end point was objective response.[84][Level of evidence C3]

Among the 247 patients who had received prior platinum-based chemotherapy, the objective response rate was 61% (95% CI, 55%–67%), and 7% achieved complete responses. The median DOR was 28.6 months (95% CI, 20.4–could not be evaluated); 49% of responses were ongoing (median follow-up, 21.2 months). The median PFS was 24.9 months; 38% of patients were alive and progression-free at the data cutoff (median follow-up, 24.7 months).

A phase I/II study (LIBRETTO-001 [NCT03157128]) enrolled patients with RET fusion−positive solid tumors. RET fusion status was determined by local molecular testing (NGS, FISH, or polymerase chain reaction assay) without central confirmation. The primary end point was objective response.[84][Level of evidence C3]

Among the 26 patients with measurable baseline CNS metastasis by the independent review committee, the intracranial objective response rate was 85% (95% CI, 65%–96%), and 27% had complete responses.

A phase I/II study (LIBRETTO-001 [NCT03157128]) enrolled patients with RET fusion−positive solid tumors. RET fusion status was determined by local molecular testing (NGS, FISH, or polymerase chain reaction assay) without central confirmation. The primary end point was objective response.[84][Level of evidence C3]

In the full safety population (n = 796), the median treatment duration was 36.1 months.

A phase I/II study (LIBRETTO-001 [NCT03157128]) enrolled patients with RET fusion−positive solid tumors. RET fusion status was determined by local molecular testing (NGS, FISH, or polymerase chain reaction assay) without central confirmation. The primary end point was objective response.[84][Level of evidence C3]

There was no significant change in the safety profile. Most adverse events were grade 1 to 2. The most common adverse events were edema, diarrhea, fatigue, dry mouth, hypertension, increased ALT and aspartate aminotransferase (AST), and rash.