Phase 12 Study Evaluating Safety and Early Effectiveness of Oral JBI-802 in Adults With Myeloproliferative Neoplasms and MyelodysplasticMyeloproliferative Neoplasms With High Platelet Counts

What this Trial Is About

Researchers are evaluating the safety and preliminary effectiveness of JBI-802, an oral drug, in adults diagnosed with Myeloproliferative Neoplasms MPN and MyelodysplasticMyeloproliferative Neoplasms MDSMPN who have thrombocytosis. This phase 12 open-label study aims to find the best dose that is safe and tolerable and then assess how well the drug works for these conditions. JBI-802 targets specific enzymes involved in gene regulation and cancer development, and has shown promising preclinical results. Participants will take JBI-802 capsules daily on an empty stomach in 28-day treatment cycles for up to 2 years, or until the drug is no longer tolerated, disease progresses, or other reasons lead to stopping treatment. The study includes a dose escalation phase using a 33 design starting at 5 mgday, with possible dose increases or decreases based on safety and tolerability. After establishing the recommended dose, an expansion phase will enroll more participants at that dose to further evaluate safety and efficacy. During the study, participants will undergo assessments including medical history reviews, physical exams, vital signs, ECGs, symptom questionnaires, imaging scans, bone marrow biopsies, and various blood and urine tests. Safety and side effects will be monitored at the start and end of each cycle, and participants will keep a dosing diary to track compliance. Follow-up visits will continue after treatment ends, and compassionate use of JBI-802 may be considered after study completion based on clinical judgment and availability.

Phase 1/ Phase 2 Study to Assess Safety and Efficacy of Orally Administered JBI-802 in Subjects With Myeloproliferative Neoplasms (MPN) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN) With Thrombocytosis