MDS/MPN Clinical Trial
Official title:
The ABNL-MARRO 001 Study: A Phase 1/2 Study of Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes.
ABNL-MARRO (A Basket study of Novel therapy for untreated MDS/MPN and Relapsed/Refractory Overlap Syndromes) is an international European-American cooperation providing the framework for collaborative studies to advance treatment of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) and explore clinical-pathologic markers of disease severity, prognosis and treatment response. ABNL MARRO 001 (AM-001) is an Open label, phase 1/2 study within the framework of the ABNL-MARRO that will test novel treatment combinations in MDS/MPN. Each Arm of AM-001 will test an active myeloid target compound in combination with ASTX727, an oral drug combining fixed doses of the DNA methyltransferase inhibitor (DNMTi) decitabine and the cytidine deaminase inhibitor E7727, also known as cedazuridine in a single tablet.
Current Arm: ASTX727 + itacitinib (INCB039110; JAK1 inhibitor) Primary Objective Phase 1: - Characterize the dose-limiting toxicities (DLTs) of each novel oral targeted agent in combination with oral ASTX727 in order to determine the recommended phase 2 dose (RP2D) and schedule. Primary Objective Phase 2: - To test whether the overall response to each novel ASTX727 combination therapy in MDS/MPN patients is sufficiently high to warrant further investigation in more definitive trials. Secondary Objectives: - To expand the safety analysis of each treatment combination in MDS/MPN patients. - To assess the morphologic bone marrow response in MDS/MPN patients treated on each Arm of the study. - To estimate the effect of each treatment combination on patient survival - To test the applicability of the proposed MDS/MPN IWG response criteria across multiple Arms of this study. Tertiary/Exploratory objectives: - To investigate genetic biomarkers of response in MDS/MPN. - To characterize molecular responses to individual treatments. - To evaluate synergistic effects of hypomethylation by ASTX727 and specific pathway blockade by study compounds. - To explore the use of automated quantification of spleen volume from CT exams as a measure of clinical benefit - To test and/or validate diagnostic algorithms and prognostic indices for MDS/MPN patients - To investigate the correlation of patient reported outcomes with disease severity and/or treatment response ;
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