Recruiting

Two medications combined to treat blood cancer that hasn't responded to prior treatment

Plain-English translation of NCT03683433 on ClinicalTrials.gov ↗ · Source last updated September 2026 · Translation generated June 2026 · 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.

What is this trial?

This trial is testing whether combining two medications—enasidenib and —can help people whose acute myeloid leukemia (a blood cancer) has stopped responding to their previous treatments. The study focuses specifically on patients whose leukemia has a particular genetic change called an IDH2 mutation. Researchers want to see if this two-drug combination can help more patients achieve remission and live longer.

Acute myeloid leukemia is difficult to treat once it comes back or stops working with standard chemotherapy, and many patients don't have good options left. This medication targets a specific genetic change found in some patients' cancer cells, and researchers believe combining it with another drug might be more effective than using either one alone.

Do you qualify?

You likely qualify if…

you have been diagnosed with acute myeloid leukemia (or a related blood cancer) that has either come back after treatment or stopped responding to prior therapy
your cancer cells have been tested and found to have an IDH2 gene mutation
you are generally able to care for yourself with minimal assistance (ECOG performance status 3 or better)
your kidney and liver function is acceptable, as measured by blood tests
you are willing to use effective birth control during the study and for 3 months after treatment ends (if you can become pregnant or father children)

You likely don't qualify if…

you have a specific type of leukemia called acute promyelocytic leukemia (APL) or carry the t(15;17) genetic change
you have an active, uncontrolled infection or serious heart condition that is not well managed with medications
you have uncontrolled high blood pressure or dangerous heart rhythm problems despite medical treatment
you are pregnant, breastfeeding, or unwilling to use contraception as required
you have other medical, psychological, or social conditions that would make it unsafe or impossible for you to participate

What participation looks like

You would receive through an injection under the skin or through an IV over 30 minutes on days 1–7 of each cycle, and take enasidenib as a pill by mouth once daily starting on day 1. Treatment cycles repeat every 4–6 weeks for as long as the cancer responds and side effects stay manageable. After your treatment ends, you would have follow-up visits at 30 days, then every 3–6 months for up to 5 years to monitor your health and cancer status.

AI-generated summary from trial data · Jun 24, 2026 · Not medical advice

Trial locations(1 site)

United States

M D Anderson Cancer Center, Houston, Texas