The ABNL-MARRO 001 Study: A Phase 1/2 Study of Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes.
Michael Savona
Summary
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.
Description
Current Arm: ASTX727 + ruxolitinib (JAK1/JAK2 inhibitor) 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 MD…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Must be ≥ 18 years of age at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and must be willing and able to meet all study requirements. 2. Must have morphologically confirmed diagnosis of MDS/MPN, excluding JMML, in accordance with WHO (2016) diagnostic criteria (Appendix 1, Section 12.1). 3. Treatment-naïve patients (patients who have had no prior disease-modifying therapy) may enroll in any AM-001 Arm that is open to accrual in phase 1 or phase 2. Treatment-naïve patients may have received recombinant erythropoietin, danazol, hydro…
Interventions
- DrugASTX727
Taken by mouth daily during days 1-5 of every 28-day cycle.
- DrugItacitinib
Taken by mouth daily during each 28-day cycle
- DrugRuxolitinib
Dosage will be 5,15, or 20mg Taken by mouth BID for 28-day cycle
Locations (4)
- Moffitt Cancer CenterTampa, Florida
- University of Rochester Wilmot Cancer InstituteRochester, New York
- Oregon Health Sciences UniversityPortland, Oregon
- Vanderbilt-Ingram Cancer CenterNashville, Tennessee