Recruiting

Testing a new cancer drug alone and combined with another medication

Plain-English translation of NCT06484062 on ClinicalTrials.gov ↗ · Source last updated June 2026 · Translation generated July 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This trial is testing a new medication called to treat two types of blood cancer: acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Researchers want to find the right dose of this medication when taken alone or combined with another drug called ASTX727, and to see if it helps patients whose cancers have returned or are no longer responding to previous treatments.

Some patients with these blood cancers stop responding to standard treatments or see their cancer come back, leaving them with limited options. This trial exists to find out whether this new medication might help these patients by working in a different way than existing treatments.

Do you qualify?

You likely qualify if…

you have been diagnosed with acute myeloid leukemia or myelodysplastic syndromes that has come back or is no longer responding to previous treatment
you are at least 18 years old
your cancer has specific genetic changes (like FLT3, IDH1, or IDH2) and you have already tried the approved drugs that target these mutations
you have tried at least one or more standard treatment regimens before enrolling, depending on which group you are in
for the combination treatment group: you have newly diagnosed high-risk myelodysplastic syndrome and have had little to no prior chemotherapy

You likely don't qualify if…

you are pregnant or breastfeeding
you have severe heart, kidney, or liver problems
you have an active infection that is not controlled
you cannot take oral medications by mouth

What participation looks like

Depending on which group you join, you will take the medication by mouth on specific days each month in 28-day cycles. You will have regular visits including blood tests, bone marrow samples, and heart imaging to check how you're responding and monitor for side effects. Your participation continues as long as the treatment is helping and you are not experiencing unacceptable side effects.

AI-generated summary from trial data · Jul 3, 2026 · Not medical advice

Testing a new cancer drug alone and combined with another medication

What is this trial?

Do you qualify?

What participation looks like

Trial locations(22 sites)