Pharmacokinetics, Safety, and Efficacy of ASTX727 in Combination With Venetoclax in Acute Myeloid Leukemia (AML)

Summary

The Phase 1 portion of this study is a single-arm, open-label, multicenter, non-randomized interventional study to evaluate the pharmacokinetic (PK) interaction, safety, and efficacy of ASTX727 when given in combination with venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy. The primary purpose of the study is to rule out drug-drug interactions between ASTX727 and venetoclax combination therapy by evaluating area under the curve (AUC) and maximum plasma concentration (Cmax) exposure. The Phase 2 portion of the study is to assess the efficacy of ASTX727 and venetoclax when given in combination and to evaluate potential PK interactions. Phase 2 will follow the same overall study design as Phase 1 and has two parts, Part A and Part B.

Timeline

Start

2021-02-09

Primary completion

2024-09-30

Completion

2026-12-31

Publications

• Roboz GJ, Zeidan AM, Mannis GN, Montesinos P, Arnan M, Savona MR, Odenike O, McCloskey JK, Amin HV, Fathi AT, Bernal Del Castillo T, Rodriguez-Macias G, Liesveld JL, Im AP, Cerny J, Gentile TC, Oganesian A, Chan D, Wan Y, Dijkstra M, Keer HN, Griffiths EA, DiNardo CD. All-Oral Treatment of Newly Diagnosed Acute Myeloid Leukemia. N Engl J Med. 2026 Jun 4;394(21):2107-2116. doi: 10.1056/NEJMoa2510223.

Drugs

Indications