Recruiting

Testing a new targeted drug for hard-to-treat blood cancers

Plain-English translation of NCT07270770 on ClinicalTrials.gov ↗ · Source last updated March 2026 · Translation generated June 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 , an oral medication designed to target a specific protein called menin in patients whose acute leukemia has come back or stopped responding to prior treatments. The medication works by turning off genes that help leukemia cells survive and grow, potentially helping cancer cells die or become sensitive to other treatments again. Researchers believe this approach may work especially well for patients whose cancers have certain genetic changes (KMT2A rearrangements or NPM1 mutations) that make them harder to treat.

People with relapsed or refractory acute leukemia — especially those with certain genetic mutations — have very limited treatment options and poor outcomes with current therapies. This medication offers a new approach by targeting a different pathway in cancer cells, addressing an urgent need for better treatments for this high-risk group.

Do you qualify?

You likely qualify if…

you are at least 16 years old
you have been diagnosed with acute leukemia that has returned or stopped responding to at least one prior treatment
you have genetic changes called KMT2A rearrangement or NPM1 mutation (preferred, though not required)
your overall health status allows you to care for yourself with minimal assistance (ECOG score 0–2)
your liver, kidney, and heart function are adequate enough to safely take the medication
you are willing to use effective birth control during the trial and for 90 days after your last dose

You likely don't qualify if…

you have leukemia that has spread to your brain or spinal cord (unless it was previously treated and is now controlled)
you have active hepatitis B, hepatitis C, or HIV infection
you have significant heart problems, including a history of heart attack, unstable chest pain, or heart failure within the past 6 months
you have had another cancer diagnosed in the past 5 years (except certain skin or breast cancers)
you received a stem cell transplant within the past 2 months or are currently having graft-versus-host disease or taking immunosuppressive medications

What participation looks like

You would take the medication by mouth twice daily in 28-day cycles, with the dose potentially increasing as the trial progresses to find the safest and most effective level. Throughout the trial, you would have regular blood tests, heart monitoring, and clinic visits to check how well the medication is working and watch for side effects. You would continue treatment until your cancer improves, side effects become too serious, you have a stem cell transplant, or your doctor decides it is no longer helping — with the entire process lasting weeks to months depending on your response.

AI-generated summary from trial data · Jun 27, 2026 · Not medical advice

Testing a new targeted drug for hard-to-treat blood cancers

What is this trial?

Do you qualify?

What participation looks like

Trial locations(1 site)