Phase I study of AR-42 and decitabine in acute myeloid leukemia

Sophia G. Liva, Christopher C. Coss, Jiang Wang, William Blum, Rebecca Klisovic, Bhavana Bhatnagar, Katherine Walsh, Susan Geyer, Qiuhong Zhao, Ramiro Garzon, Guido Marcucci, Mitch A. Phelps, Alison R. Walker

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


This phase I trial sought to determine a biologically safe and effective dose of AR-42, a novel histone deacetylase inhibitor, which would lead to a doubling of miR-29b prior to decitabine administration. Thirteen patients with previously untreated or relapsed/refractory AML were treated at 3 dose levels (DL): AR-42 20 mg qd on d1,3,5 in DL1, 40 mg qd on d1,3,5 in DL2 and 40 mg qd on d1,3,4,5 in DL3. Patients received decitabine 20 mg/m2 on d6–15 of each induction cycle and 20 mg/m2 on d6–10 of each maintenance cycle. One DLT of polymicrobial sepsis and multi-organ failure occurred at DL3. Two patients achieved a CRi and one patient achieved a CR for an ORR of 23.1%. The higher risk features of this patient population and the dosing schedule of AR-42 may have led to the observed clinical response and failure to meet the biologic endpoint.

Original languageEnglish (US)
Pages (from-to)1484-1492
Number of pages9
JournalLeukemia and Lymphoma
Issue number6
StatePublished - May 11 2020


  • AR-42
  • Acute myeloid leukemia
  • HDAC inhibitor
  • REC-2282
  • decitabine
  • miR-29b

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


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