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Experimental combo targets tough blood cancers

NCT ID NCT05636514

First seen Jan 05, 2026 · Last updated Jun 19, 2026 · Updated 24 times

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.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Concord Repatriation and General Hospital

    RECRUITING

    Sydney, New South Wales, 2139, Australia

    Contact

  • Nepean Hospital

    RECRUITING

    Sydney, New South Wales, 2747, Australia

    Contact

  • Prince of Wales Hospital

    RECRUITING

    Sydney, New South Wales, 2031, Australia

    Contact

  • Royal Prince Alfred Hospital

    RECRUITING

    Sydney, New South Wales, 2050, Australia

    Contact

  • Westmead Hospital

    RECRUITING

    Westmead, New South Wales, 2145, Australia

    Contact

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.

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.