Phase II study of CC-486 (oral azacitidine) in previously treated patients with locally advanced or metastatic nasopharyngeal carcinoma

Ricard Mesia, Paolo Bossi, Aaron R. Hansen, Ching Yun Hsieh, Lisa F. Licitra, Eng Huat Tan, Peng Chen, Julie Ann Miller, Lilian L. Siu, Robert I. Haddad

Research output: Contribution to journalArticlepeer-review


Background: Treatment options are limited for recurrent nasopharyngeal carcinoma (NPC). We report results from a phase II study of CC-486 (oral azacitidine) in advanced NPC. Patients and methods: Patients with locally advanced or metastatic NPC and 1–2 prior treatment regimens received CC-486 300 mg daily on days 1–14 of 21-day cycles until disease progression or unacceptable toxicity. The first 6 patients of Asian-Pacific Islander (API) ethnicity received a reduced dose of 200 mg to preserve safety and tolerability; if well tolerated, subsequent API patients received CC-486 300 mg. The study could advance to stage 2 if > 4 patients achieved a response. Co-primary end-points were overall response rate (ORR) and progression-free survival (independent review). Key secondary end-points were overall survival and safety. Results: Owing to faster-than-anticipated enrolment, 36 patients, including 13 of API ethnicity, were enrolled; the median age was 54.0 years. Most patients were male (81%) and had an Eastern Cooperative Oncology Group performance status ≤ 1 (97%). Among 25 efficacy-evaluable patients, the ORR was 12%; the median progression-free and overall survival were 4.7 and 18.0 months, respectively. The most common grade III/IV treatment-emergent adverse events were neutropenia (33%) and febrile neutropenia (11%). Twenty-one posttreatment deaths, primarily due to progressive disease or disease complications, and 1 on-treatment death (epistaxis, unrelated to study drug) occurred. The study did not advance to stage 2. Conclusion: CC-486 did not show sufficient clinical activity to support further development as monotherapy in this patient population. The safety profile of CC-486 in NPC was consistent with that in other solid tumours.

Original languageEnglish
Pages (from-to)138-145
Number of pages8
JournalEuropean Journal of Cancer
Publication statusPublished - Dec 2019


  • Azacitidine
  • CC-486
  • Epigenetic
  • Nasopharyngeal carcinoma
  • Pharmacokinetics

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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