Background: Second/third-line treatment (Tx) options are limited for pts with NPC. The efficacy and safety of CC-486 were assessed using Simon's optimal 2-stage design in a phase 2 study. The predefined criterion for advancement to stage 2 (> 4 complete/ partial responses) was not met. Here, we present final results from stage 1. Methods: The single-arm, open-label study (NCT02269943) included pts with locally advanced/metastatic NPC who had ≥1 prior Tx, including ≥1 platinum-containing regimen. Pts received CC-486 300 mg orally on d 1-14 of a 21-d cycle until disease progression/ unacceptable toxicity. The first 6 Asian-Pacific (AP) pts received CC-486 200 mg; if well tolerated, subsequent AP pts received 300 mg. Primary endpoints (per independent reviewer assessment): overall response rate, progression-free survival. Key secondary endpoints: overall survival, disease control rate, safety, pharmacokinetics (PK). Results: Median age of 36 enrolled pts was 54.0 y. Most were male (81%), had ECOG PS of≤1 (97%), and had ≥2 prior systemic anticancer Tx (58%); 36% were AP. Pts received a median of 7.0 Tx cycles; 44% had ≥1 dose interruption and 39% had ≥ 1 dose reduction. 22 pts died: 1 on- and 21 post-Tx (> 28 d after last dose); 18 died due to disease complications. The table shows efficacy outcomes. PK analysis showed rapid absorption; exposure was comparable between doses. However, large inter-pt variability and small pt numbers did not allow definitive conclusions. All 36 safety-evaluable pts had ≥ 1 Tx-emergent adverse event (TEAE). Common TEAEs included vomiting (72%) and nausea (67%). Common grade 3/4 TEAEs included neutropenia (33%) and febrile neutropenia (11%). Conclusions: The safety profile of CC-486 in pts withNPC was consistent with that in other solid tumors and of azacitidine. CC-486monotherapy did not show sufficient clinical activity in the selected pt population to support further development in these pts. (Table Presented) .
CITATION STYLE
Mesia Nin, R., Bossi, P., Hansen, A., Hsieh, C., Licitra, L. F., Tan, E. H., … Haddad, R. (2018). Phase II study of CC-486 in previously treated patients (pts) with locally advanced/metastatic nasopharyngeal cancer (NPC): Final results. Annals of Oncology, 29, viii382. https://doi.org/10.1093/annonc/mdy287.027
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