Could a pill replace shots for MDS? early trial begins

NCT ID NCT04608110

Recruiting now Disease control Sponsor: Taiho Pharmaceutical Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Aug 11, 2026 · Updated 3 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

MYELODYSPLASTIC SYNDROME (MDS)

Contacts and locations

Locations

Fukushima Medical University Hospital
RECRUITING

Fukushima, Japan
Kindai University Hospital
RECRUITING

Sakai, Japan
NTT Medical Center Tokyo
RECRUITING

Shinagawa-Ku, Japan
Nagasaki University Hospital
RECRUITING

Nagasaki, Japan
Nippon Medical School Hospital
RECRUITING

Bunkyō City, Japan
Osaka City General Hospital
RECRUITING

Osaka, Japan
Saitama Medical University Hospital
RECRUITING

Iruma, Japan
Tokyo Medical University Hospital
RECRUITING

Shinjuku-Ku, Japan
University Hospital, Kyoto Prefectural University of Medicine
RECRUITING

Kyoto, Japan
Yamagata University Hospital
RECRUITING

Yamagata, Japan

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