Could a pill replace shots for MDS? early trial begins

NCT ID NCT04608110

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial is testing an oral form of the cancer drug azacitidine (called ASTX030) for people with myelodysplastic syndrome (MDS). The goal is to see if the pill version reaches similar levels in the blood as the standard injection. About 40 adults with MDS will take part to help find the right dose.

What this could mean

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

Active substance

ASTX030 (oral azacitidine plus cedazuridine)

What this could lead to

If successful, this could provide a more convenient oral treatment option for MDS, potentially replacing injections.

What could go wrong

This is a very early Phase 1 trial with only 40 people, focused on dosing, not on proving the drug works. The oral version may not work as well as the injection.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

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

Locations

  • Fukushima Medical University Hospital

    RECRUITING

    Fukushima, Japan

    Contact Phone: •••-•••-••••

  • Kindai University Hospital

    RECRUITING

    Sakai, Japan

    Contact Phone: •••-•••-••••

  • NTT Medical Center Tokyo

    RECRUITING

    Shinagawa-Ku, Japan

    Contact Phone: •••-•••-••••

  • Nagasaki University Hospital

    RECRUITING

    Nagasaki, Japan

    Contact Phone: •••-•••-••••

  • Nippon Medical School Hospital

    RECRUITING

    Bunkyō City, Japan

    Contact Phone: •••-•••-••••

  • Osaka City General Hospital

    RECRUITING

    Osaka, Japan

    Contact Phone: •••-•••-••••

  • Saitama Medical University Hospital

    RECRUITING

    Iruma, Japan

    Contact Phone: •••-•••-••••

  • Tokyo Medical University Hospital

    RECRUITING

    Shinjuku-Ku, Japan

    Contact Phone: •••-•••-••••

  • University Hospital, Kyoto Prefectural University of Medicine

    RECRUITING

    Kyoto, Japan

    Contact Phone: •••-•••-••••

  • Yamagata University Hospital

    RECRUITING

    Yamagata, Japan

    Contact Phone: •••-•••-••••