Experimental combo targets tough blood cancers

NCT ID NCT05636514

Recruiting now Disease control Sponsor: Clinical Hub for Interventional Research (CHOIR) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial is testing whether combining two drugs—decitabine/cedazuridine (already approved for MDS) and the experimental drug defactinib—is safe and tolerable for people with high-risk myelodysplastic syndromes, certain acute myeloid leukemias, and chronic myelomonocytic leukemia. Twelve participants will receive the combination for up to six cycles. The main goal is to find the highest safe dose and monitor side effects.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: decitabine/cedazuridine (ASTX727) and defactinib (VS-6063)
What this could lead to: If this combination is safe and effective, it could offer a new treatment option for people with high-risk MDS, AML, or CMML.
What could go wrong: This is a very early phase 1 trial with only 12 participants, so it is mainly testing safety and dosing. The combination may not work better than existing treatments, and side effects could be serious.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia chronic myelomonocytic leukemia myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ACUTE MYELOID LEUKEMIA CHRONIC MYELOMONOCYTIC LEUKEMIA MYELODYSPLASTIC SYNDROMES

Contacts and locations

Locations

Concord Repatriation and General Hospital
RECRUITING

Sydney, New South Wales, 2139, Australia
Nepean Hospital
RECRUITING

Sydney, New South Wales, 2747, Australia
Prince of Wales Hospital
RECRUITING

Sydney, New South Wales, 2031, Australia
Royal Prince Alfred Hospital
RECRUITING

Sydney, New South Wales, 2050, Australia
Westmead Hospital
RECRUITING

Westmead, New South Wales, 2145, Australia

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