CDFV890G12101:A phase 1/1b, open label, multi-center, dose escalation and dose expansion study to assess the safety and efficacy of DFV890 as single agent and in combinations in adult patients with myeloid diseases

The purpose of this trial is to find an optimal dose of DFV890 that can be given to adults with myeloid diseases. Myeloid diseases affect production of normal blood cells by causing inflammation and cell death in the bone marrow. As a result, lower levels of normal blood cells are presented in patients with myeloid diseases. DFV890 is a drug designed to lower inflammation by blocking a protein called NLRP3 that can trigger inflammation.This trial is designed to find out if DFV890 can lower inflammation from myeloid diseases and increase levels of normal blood cells. The trial is also designed to learn more about the safety of DFV890.The DFV890G12101 study is an open-labeled multicentered clinical trial meaning that the patient, investigators, study teams, and Novartis will know the treatment assigned. About 80 adult patients will take part in the trial for 24 weeks, or longer if patients have clinical benefits from DFV890. The first part of the study will test 2 doses of DFV890 (25 mg and 50 mg; both taken twice daily by mouth) and determine the optimal dose. Once an optimal dose is determined, the second part of the study will continue to test the optimal dose in a larger group of patients with Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) to further understand DFV890.For this study, days are organised into a group called a cycle. Each cycle is 28 days. Patients will be asked to come to the hospital four times during the first cycle and then once during each cycle after Cycle 1. Patients in the study may not directly benefit from their participation, but Novartis may learn new information about the disease that could help treat other patients in the future. The study is organised/funded by Novartis Pharma AG and run by medical staff in hospital.