First-In-Human Study Evaluating Safety, Dosage, and Early Effects of ABD-3001 in Adults with Relapsed or Refractory Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndromes Ineligible for Intensive or Targeted Therapy

What this Trial Is About

Researchers are evaluating ABD-3001, a new drug, in adult patients with relapsed or refractory Acute Myeloid Leukemia (AML) and high-risk Myelodysplastic Syndromes (MDS) who are not eligible for intensive or new targeted therapies. This first-in-human, open-label Phase 1 study aims to understand the safety, tolerability, pharmacokinetics, and early signs of activity of ABD-3001 through two parts: a Single Ascending Dose (SAD) phase and a Multiple Ascending Dose (MAD) phase. In the SAD phase, patients receive a single fixed 4-hour intravenous infusion of ABD-3001 at increasing doses, starting from 18 mg/m² up to 540 mg/m², to identify the maximum tolerated dose and suitable dosing frequency. The MAD phase uses the data from SAD to select up to three dose levels, administered once or twice weekly over three cycles of 28 days each, to refine the dose range and administration schedule. The study is conducted at multiple centers and includes dose escalation and optimization. Participants undergo various assessments including safety and tolerability monitoring, pharmacokinetic and pharmacodynamic evaluations, and preliminary efficacy measurements during both study phases. The SAD part lasts 7 days, while the MAD part extends over 3 months. Researchers closely observe patient responses to determine recommended doses for future trials while monitoring for any adverse effects throughout the study period.

First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients