New pill shows promise against tough blood cancers
NCT ID NCT04167917
First seen Jun 27, 2026 ยท 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 โ 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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ACUTE MYELOID LEUKEMIA are added.
By submitting, you agree to our Terms of use
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
Locations
-
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
-
University of Alabama at Birmingham
Birmingham, Alabama, 35294, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a pill keep blood cancer at bay after a stem cell transplant?
- Could muscle loss predict blood cancer outcomes?
- Can a modified enzyme starve Hard-to-Treat blood cancers?
- Can a Low-Dose antifungal shield vulnerable patients from deadly fungal infections?
- Can a gentler chemo prep make stem cell transplants safer for blood cancer patients?
- Talking about the end: could guided conversations reduce futile cancer treatments?