Phase 1 Study of Oral PCLX-001 to Find the Safe Dose in Adults With Relapsed or Refractory Acute Myeloid Leukemia

What this Trial Is About

Researchers are studying oral zelenirstat PCLX-001 in patients with relapsed or refractory acute myeloid leukemia RR AML to find the safest and most effective dose. This Phase 1 trial includes two parts Dose Escalation to find the minimum safe and biologically effective dose, and Dose Expansion to further evaluate safety and preliminary activity at that dose. Patients have previously received treatment for AML and are unable to benefit from other therapies. The study involves daily oral doses of zelenirstat taken in 28-day cycles, starting at 40 mg and increasing through several dose levels up to 280 mg. In Dose Escalation, small groups of patients receive ascending doses to monitor safety and drug effects, with adjustments based on observed toxicities. Dose Expansion will treat about 20 patients at the determined safe and effective dose to assess further safety and clinical response. Participants will be monitored through regular evaluations including toxicity checks, pharmacokinetic and pharmacodynamic assessments, and laboratory tests to track liver, kidney, and heart function. Their ability to take oral medication and use contraception is required. Researchers will measure the recommended dose for expansion and overall clinical responses over approximately 18 months. The study started in March 2025 and follows patients until September 2026.

Study of Oral PCLX-001 in R/R Acute Myeloid Leukemia