Table 3.
AEs leading to a change in pexidartinib (by preferred term) reported in Phase 2 (≥9% occurrence in all patients in Phase 2), the combined RP2D groups in Phase 1b and Phase 2 (mITT RP2D population), and the study overall (mITT Population)
| AEa | Combination therapy dose/adjuvant therapy doseb | Phase 2 total N = 43 |
Combined RP2Dc Phases 1b and 2 N = 53 5 days/varies |
Study overall N = 65 |
|
|---|---|---|---|---|---|
| 800 mg/800 mg N = 27 5 days/7 days |
800 mg/None N = 16 5 days/None |
||||
| Any event | 16 (59) | 9 (56) | 25 (58) | 33 (62) | 39 (60) |
| Neutropenia | 5 (19) | 1 (6) | 6 (14) | 7 (13) | 7 (11) |
| Alanine aminotransferase increased | 1 (4) | 4 (25) | 5 (12) | 5 (9) | 6 (9) |
| Aspartate aminotransferase increased | 0 | 4 (25) | 4 (9) | 5 (9) | 6 (9) |
| Platelet count decreased | 2 (7) | 1 (6) | 3 (7) | 5 (9) | 6 (9) |
| Thrombocytopenia | 2 (7) | 1 (6) | 3 (7) | 5 (9) | 7 (11) |
Abbreviations: mITT = modified intention-to-treat population; N = number of patients; RP2D = recommended Phase 2 dose.
aAEs with pexidartinib action taken of “drug temporarily withdrawn,” “drug permanently withdrawn,” or “dose reduced” are summarized.
bPatients are summarized by doses of pexidartinib taken during combination therapy and adjuvant therapy periods. Patients reported as having discontinued the study before adjuvant treatment are summarized as having adjuvant treatment of “none.”
cThe combined RP2D group includes all patients in Phase 1 or Phase 2 who received 800 mg, 5 days/week of pexidartinib during combination therapy. There were 10 patients from Phase 1b and 43 patients from Phase 2.