Phase 1 Study of Rebecsinib Infusion for Adults with Relapsed or Refractory Secondary Acute Myeloid Leukemia or High-Risk Myelofibrosis

What this Trial Is About

Researchers are evaluating the safest and most effective dose of rebecsinib, an investigational drug, for patients with relapsed or refractory secondary acute myeloid leukemia sAML or higher-risk myelofibrosis MF. This study is designed as a Phase 1 trial focusing on treatment, aiming to find the maximum tolerated or biologically active dose and assess safety by monitoring adverse events in these patient groups. Participants receive rebecsinib by intravenous infusion on Days 1, 4, 8, and 11 of each 28-day cycle for a total of six cycles. The study tests various dose levels based on ideal body weight, ranging from 0.2 to 8.0 mgkg. The treatment is given sequentially, with careful dose escalation to determine the best tolerated dose. During the study, participants will be closely monitored for safety and effectiveness through clinical assessments, adverse event tracking, and measurements of response rates, progression-free survival, and overall survival. Pharmacokinetics and pharmacodynamics of rebecsinib will also be explored. The primary outcome measures include determining the maximum tolerated dose over a period of about two years and evaluating treatment-emergent adverse events for approximately four months after enrollment.

A Study of Rebecsinib for Patients With Relapsed/Refractory Secondary Acute Myeloid Leukemia or High Risk Myelofibrosis