Recruiting

Testing a new protein blocker for blood cancers

Plain-English translation of NCT07064122 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 is an early-stage trial testing a new medication called , which blocks a protein in the body called IRAK4. The trial will evaluate whether this medication is safe and effective for people with certain blood cancers (specifically MDS and CMML) that have come back or stopped responding to prior treatments.

People with certain blood cancers often run out of effective treatment options after their disease stops responding to standard therapies. This trial exists to see if blocking the IRAK4 protein might offer a new way to treat these hard-to-treat blood cancers.

Do you qualify?

You likely qualify if…

you have been diagnosed with MDS (myelodysplastic syndrome) or CMML (a type of blood cancer) that came back or stopped responding to prior treatments
you have received at least one previous line of treatment for your blood cancer
you have fewer than 20% abnormal blasts (immature blood cells) in your blood or bone marrow
you are in reasonably good health (ECOG performance status of 2 or better)
you have disease symptoms that need treatment and can be measured objectively
you are willing to provide bone marrow samples before and during the study

You likely don't qualify if…

you have previously taken an IRAK inhibitor or similar protein-blocking drug
you received cancer treatment (other than hydroxyurea) within 15 days before starting the study
you have had major surgery or are still recovering from surgery within the last 28 days
you are currently taking medications that could interfere with how your body processes this medication or that affect your heart rhythm
you are pregnant, breastfeeding, or unwilling to use contraception during the trial

What participation looks like

You will take as a pill once daily at home, continuing as long as the treatment is working and you tolerate it well. The trial includes screening visits, regular 28-day treatment cycles with check-ups and blood tests, and a follow-up period 30 days after your last dose. You'll also need to provide a bone marrow sample at the beginning of the study to confirm your diagnosis.

AI-generated summary from trial data · Jun 1, 2026 · Not medical advice

Trial locations(21 sites)

United States

Research Site, Miami, Florida

Research Site, Tampa, Florida

Research Site, Houston, Texas

Australia

Research Site, Heidelberg

Research Site, Melbourne