First-In-Human, Open Label, Dose Escalation Study to Evaluate Safety, PK and PD of ABD-3001 As Monotherapy in Relapsed/Refractory Acute Myeloid Leukemia or High/Very-high Risk Myelodysplastic Syndromes Patients, Ineligible for Intensive or New Generation Targeted Therapy.

What this Trial Is About

Researchers are conducting a first-in-human, open-label, multicenter Phase 1 study to evaluate ABD-3001 in adults with relapsed or refractory Acute Myeloid Leukemia (AML) or high-risk Myelodysplastic Syndromes (MDS) who are not eligible for intensive or new targeted therapies. The study includes patients with high or very high risk based on prognostic scoring and aims to explore safety, tolerability, pharmacokinetics, and preliminary signs of activity of ABD-3001 in these difficult-to-treat blood cancers. The study has two parts: a Single Ascending Dose (SAD) phase and a Multiple Ascending Dose (MAD) phase. In the SAD phase, participants receive a single 4-hour intravenous infusion of ABD-3001 at increasing doses to find the maximum tolerated dose and appropriate dosing schedule. The MAD phase follows, where selected doses from SAD are given once or twice weekly over three 28-day cycles to evaluate repeated dosing effects. Dose escalation uses a 3+3 design, and three dosing regimens run in parallel during MAD. Participants will be monitored through safety assessments, pharmacokinetic and pharmacodynamic evaluations, and response measurements including overall response rate and survival rates over the treatment period. The study will last seven days for SAD and three months for MAD, with quality of life also assessed. Researchers will carefully track side effects and drug behavior in the body to determine safe and effective dose ranges for future studies.

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