Can a new oral drug take on hard-to-treat leukaemia?
NCT ID NCT07723703
First seen Jul 23, 2026 · Last updated Jul 24, 2026 · Updated 1 time
Summary
This early-stage trial is testing an experimental drug called AMX-883, taken by mouth, for people with acute myeloid leukaemia (AML) or high-risk myelodysplastic syndrome (MDS) that has come back or not responded to standard treatments. The study will first test AMX-883 alone, then in combination with another drug (posaconazole) in people with AML. The main goals are to check the drug's safety, find the best dose, and get an early look at whether it can help control the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- an experimental oral drug called AMX-883, sometimes given with the antifungal posaconazole
- What this could lead to
- If safe and effective, AMX-883 could offer a new treatment option for people with hard-to-treat leukaemia or related blood disorders.
- What could go wrong
- This is an early phase 1 trial with only 54 participants, focused on safety and dosing. It is too soon to know if the drug will work, and side effects are unknown.
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 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 54 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Sep 2026
An estimate. Start dates often move.
- Expected to finish
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Apr 2029
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: * Participants with relapsed or refractory AML who have failed all available standard therapies or relapsed or refractory high-risk MDS with BM blasts 10-19% * Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 * Adequate washout from prior therapies * Adequate kidney and liver function * Female participants of childbearing potential must use highly effective contraception, and male participants must agree to use barrier contraception and avoid sperm donation for at least 120 days after last dose * If enrolled in M1B: Participant must have no documented contraindication to treatment with posaconazole before start of treatment Exclusion Criteria: * Diagnosis of acute promyelocytic leukaemia or chronic myelogenous leukaemia in blast crisis * Clinically active central nervous system (CNS) leukaemia * Receiving immunosuppressive therapy post HSCT * History of another malignancy that is active, progressing, or has required systemic treatment within the past 2 years * Presence of \>Grade 1 active graft versus host disease within 4 weeks prior to C1D1 * Significant cardiovascular disease * Family history of sudden cardiac death before 40 years of age or a family history of long QT syndrome * Clinically significant electrolyte imbalances (e.g., hypokalaemia, hypomagnesaemia, hypocalcaemia) that may contribute to QT interval prolongation * Clinically significant bradycardia (\<50 beats per minute) that is symptomatic or causes haemodynamic instability * Major surgery within 4 weeks prior to C1D1 or inadequate recovery from prior surgery * Uncontrolled intercurrent illness * Inability to fast, swallow, ingest, or absorb oral medication due to a pre-existing condition * History of interstitial lung disease or pneumonitis requiring systemic corticosteroid treatment * Requirement for medications with a known risk of Torsades de Pointes that cannot be discontinued prior to study treatment * Detectable human immunodeficiency virus (HIV) viral load * Known serologic status reflecting active hepatitis B or C infection * Active uncontrolled systemic fungal, bacterial, viral, or other infection or a condition predisposing to severe infection
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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 official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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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.
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