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
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.
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.
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
To investigate the correlation of patient reported outcomes with disease severity and/or
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.