A Study of JNJ-74856665 in Participants With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

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This is first-in-human (FIH) Phase 1), dose escalation study of JNJ-74856665 alone or in combination with Azacitidine in participants with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). AML is a heterogeneous disease characterized by uncontrolled clonal expansion of hematopoietic pr...

This is first-in-human (FIH) Phase 1), dose escalation study of JNJ-74856665 alone or in combination with Azacitidine in participants with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). AML is a heterogeneous disease characterized by uncontrolled clonal expansion of hematopoietic progenitor cells (myeloid blasts) in the peripheral blood, bone marrow, and other tissues and is the second most common form of leukemia. MDS are a heterogeneous group of malignant hematopoietic stem cell disorders that are characterized by cytopenias, myeloid dysplasia, and a risk of transformation to AML. JNJ-74856665 is an orally bioavailable, potent, and selective dihydroorotate dehydrogenase (DHODH) inhibitor that binds to the enzyme's ubiquinone binding site promoting AML/MDS differentiation as well as cell cycle arrest and apoptosis. Azacitidine (5-azacytidine) is a nucleoside metabolic inhibitor that been US Food and Drug Administration-approved for several MDS subtypes. The study is divided into 3 periods: a Screening Phase (within 28 days before the first dose of study drug), a Treatment Phase (first dose of study drug until the completion of the end-of-treatment visit) and a Post-treatment Follow-up Phase (up to the end of study participation or end of study). The end of study is defined as the last study assessment for the last participant in the study. Total duration of study is up to 3.7 years. Efficacy, safety, pharmacokinetics (PK), and biomarkers will be assessed at specified time points during this study. Participants safety and study conduct will be monitored throughout the study.

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