Phase 1 Study of Oral GLB-001 to Evaluate Safety and Effectiveness in Adults with Relapsed or Refractory Myeloid Malignancies

What this Trial Is About

Researchers are evaluating GLB-001, an oral drug, in patients with relapsed, refractory, or intolerant myeloid malignancies including polycythemia vera, essential thrombocythemia, myelofibrosis, various myelodysplastic syndromes, and acute myeloid leukemia. This phase 1, open-label study aims to assess the safety, tolerability, how the drug moves through and affects the body, and early signs of efficacy. The study includes three parts dose escalation, dose exploration, and dose expansion to find the best dose for future studies. The study involves giving GLB-001 orally to participants in three phases. Phase 1a uses a dose-escalation design to evaluate safety and tolerability in patients with polycythemia vera or essential thrombocythemia. Phase 1b also uses dose escalation to study safety in patients with myelofibrosis, myelodysplastic syndromes, and acute myeloid leukemia. Phase 1c expands dosing to further assess tolerability, efficacy, and to select the recommended dose for phase 2 trials. Dose levels and treatment schedules are adjusted based on participant response and safety. Participants will undergo assessments to monitor dose-limiting toxicities within 28 days of the first dose and longer-term safety up to three years. Researchers will evaluate drug levels in the body and clinical responses to treatment over periods ranging from weeks to years depending on the disease type and study phase. Major organ functions, blood tests, adverse events, and response to treatment will be closely monitored throughout the study. Participants must agree to follow the visit schedule and study requirements during their involvement.

A Study of GLB-001 in Patients With Myeloid Malignancies