New pill shows promise against tough blood cancers
NCT ID NCT04167917
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tested an oral drug called NTX-301 in 22 adults with certain blood cancers (MDS, AML, or CMML) that had not responded to prior treatments. The main goal was to check safety and find the right dose. Researchers also looked for signs that the drug might help control the disease. Because this is a first-in-human study, results are preliminary and will guide future research.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
NTX-301 (an oral drug that targets a protein called DNMT1 to potentially slow cancer growth)
What this could lead to
If this drug proves safe and effective in larger trials, it could offer a new oral treatment option for people with certain blood cancers.
What could go wrong
This is a very early, small Phase 1 trial with only 22 participants, so results may not apply broadly. The drug may cause side effects or fail to show meaningful benefit.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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University of Alabama at Birmingham
Birmingham, Alabama, 35294, United States