Plain-English translation of NCT04493164 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing whether combining two medications — CPX-351 and — can help treat a specific type of blood cancer called acute myeloid leukemia (AML) or a related condition called high-risk myelodysplastic syndrome (MDS), but only in patients whose cancer cells have a particular genetic change called an IDH1 mutation. The trial will look at how well the medication works and how safe it is when the two drugs are given together.
People with this specific genetic mutation in their blood cancer have limited treatment options, and doctors want to see if combining these two medications might work better together than either one alone to help patients go into remission and stay healthy longer.
You likely qualify if…
You likely don't qualify if…
If you qualify, you would start with an induction phase where you receive CPX-351 through an IV infusion on certain days and take as an oral pill daily for about a month. If your cancer goes into remission, you move to a consolidation phase with similar medications but on a modified schedule for up to 2 months. After that, you would take the oral medication alone for maintenance therapy for up to 2 years. Throughout the trial, you would have regular blood tests, check-ups, and monitoring visits, followed by continued follow-up appointments for 3 years after treatment ends.
AI-generated summary from trial data · Aug 31, 2026 · Not medical advice
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