Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS

The goal of this project is to see if two new potential treatments (defactinib and the combination tablet of decitabine/cedazuridine) can safely be combined to improve outcomes in people with high-risk myelodysplastic syndrome (MDS), certain forms of Acute Myeloid Leukaemia (AML), and Chronic Myelomonocytic Leukaemia (CMML). Decitabine/cedazuridine is approved for use by the Australian Therapeutics Goods Administration (TGA) as treatment for MDS. Defactinib is an experimental treatment. This means it is not an approved treatment for MDS in Australia. So far it has been given to over 625 patients in studies across the world. All study participants will receive active treatment, there is no placebo. Participants will take the decitabine/cedazuridine treatment once a day for 5 days in a row (day 1 to day 5) on its own for the first month (cycle). From month 2 participants will take the decitabine/cedazuridine treatment and will also take the defactinib treatment, both for 5 days in a row on days 1 to day 5 each month (cycle). Defactinib is taken twice a day.

NCT Number	NCT05636514
Lead Sponsor	Clinical Hub for Interventional Research (CHOIR) Collaborators: The University of New South Wales, Australian National University
Conditions	Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Acute Myeloid Leukemia
Enrollment	12 participants
Start Date	2022-12-14
Primary Completion	2026-08-30 (estimated)
Study Completion	2027-12-30 (estimated)
Updated on ClinicalTrials.gov	2026-03-18