New hope for tough leukemia? tiny trial tests targeted pill
NCT ID NCT07604064
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests an oral drug called olutasidenib in 3 Japanese patients whose acute myeloid leukemia (AML) has a specific IDH1 gene mutation and has not responded to or returned after other treatments. The main goal is to check safety, but researchers will also see if the drug can bring about a remission. Because it is a very small, early-phase trial, the results are preliminary and may not apply to all patients.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Olutasidenib (a targeted drug taken by mouth)
- What this could lead to
- If it works, this could point toward a treatment option for people with a specific genetic type of leukemia that has come back or not responded to other therapies.
- What could go wrong
- This is a very small, early-phase study with only 3 Japanese participants, so results may not apply broadly. The drug may not work or could cause side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 3 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jun 2026
An estimate. Start dates often move.
- Expected to finish
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Sep 2030
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Japanese patients who personally provide written informed consent to participate in this clinical trial * Patients with a confirmed diagnosis of AML based on WHO classification (2022 edition) (except acute promyelocytic leukemia with t (15:17) translocation) * Patients with relapsed or refractory AML who may or may not have undergone allogeneic hematopoietic stem cell transplantation. * Patients with IDH1 gene mutation confirmed by central confirmation after relapse or refractoriness Exclusion Criteria: * Patients with IDH2 mutations or patients with a history of IDH2 inhibitor treatment * Patients who are intolerant to IDH1 inhibitors * Patients who are deemed inappropriate for the clinical trial by the investigator or sub-investigator
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Relapsed or refractory IDH1 mutation-positive acute myeloid leukemia are added.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Research Site
RECRUITINGTokyo and Other Japanese Cities, Japan