Summary
Background
High risk myelodysplasia (MDS) is a challenging condition with limited treatment options. Patients often depend on blood transfusions and unfortunately chemotherapy is rarely successful as a treatment option. The main type of therapy used in MDS are hypomethylating agents (HMAs) such as azacitadine and decitabine. Traditionally, combination therapy has not proven to be effective in MDS given the increased toxicity of drugs used in combination with azacitidine, even where the overall responses may have been promising.
This is a component (Domain 1) study associated with master protocol registered as ACTRN12622000410752 on the Australian New Zealand Clinical Trials Registry.
Who is it for?
You may be eligible to join this study if you are aged 16 years or above, and have a diagnosis of MDS or acute myeloid leukaemia (AML) with <30% blasts.
What this trial involves
This clinical trial aims to test a relatively non-cytotoxic drug called ES-3000, in combination with a HMA named ASTX727. All participants will receive treatment in 28-day cycles.
At first, each participant will receive a single cycle of ES-3000 by itself, in order to take blood samples at various timepoints throughout the day to establish how the drug is metabolised by the body. ES-3000 is an oral tablet administered three times per day on days 1-14 of the treatment cycle. Each subsequent participant recruiting to the trial will receive a higher dose of ES-3000, until the dose is no longer tolerated.
From the second cycle onwards, participants will receive the same dose of ES-3000 in combination with a previously established safe dose of ASTX727 once per day on days 1-5 of each treatment cycle. This will continue until the participant experiences either unacceptable toxicity or disease progression.
Participants will be monitored for adverse effects for the duration of treatment, as well as every 3 months for assessment of their response to treatment through blood samples and bone marrow biopsies, and assessment of quality of life through completion of a questionnaire.