Experimental drug for tough leukemias hits dead end

NCT ID NCT03922100

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-phase trial tested an oral drug called NMS-03592088 in 63 adults with acute myeloid leukemia (AML) or chronic myelomonocytic leukemia (CMML) that had come back or not responded to standard treatments. The goal was to check safety, find the right dose, and see if it could shrink the cancer. The study was terminated early, so results are limited.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
NMS-03592088 (a targeted drug that blocks FLT3, KIT, and CSF1R proteins)
What this could lead to
If it had worked, this drug could have offered a new treatment option for people with hard-to-treat AML or CMML.
What could go wrong
The trial was terminated early, so we don't know if the drug is safe or effective. It was a small, early-phase study, and many such drugs fail to show benefit.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

63 people

The number who actually took part.

Started

Apr 2019

Finished

Aug 2024

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients with relapsed/refractory disease who have failed standard therapy or are unsuitable for standard treatment, with one the following confirmed diagnosis: AML as defined by the European LeukemiaNet (ELN) * Patients with confirmed diagnosis of AML as defined by the 2022 ELN recommendations * Patients must have failed standard of care. * Adult (age ≥ 18 years) patients * Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 * The interval from prior antitumor treatment to time of NMS-03592088 administration should be at least 2 weeks for any agents other than hydroxyurea. * All acute toxic effects (excluding alopecia) of any prior therapy must have resolved to NCI CTCAE version 5.0 Grade ≤1 * Adequate hepatic and renal function * Patients must use highly effective contraception. * Signed and dated IEC or IRB-approved informed consent form. Exclusion Criteria: * Current enrollment in another interventional clinical study * Diagnosis of acute promyelocytic leukemia or Breakpoint cluster region-Abelson (BCR-ABL)-positive leukaemia * Currently active second malignancy, except for adequately treated basal or squamous cell skin cancer and/or cone biopsied in situ carcinoma of the cervix uteri and/or superficial bladder cancer. * Patients with known leukemia involvement of central nervous system (CNS) * Hematopoietic stem cell transplantation (HSCT) within 3 months of treatment start and/or persistent non-hematologic toxicities of Grade ≥2 related to the transplant * Active acute or chronic graft versus host disease (GVHD) requiring immunosuppressive treatment * Patients with QTcF interval ≥ 480 milliseconds or with risk factors for torsade de pointes * Pregnancy. * Breast-feeding or planning to breast feed during the study or within 3 months after study treatment. * Any of the following in the previous 6 months: myocardial infarction, unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident or transient ischemic attack, pulmonary embolism, deep vein thrombosis * Known active, life threatening or clinically significant uncontrolled systemic infection. * Known active gastrointestinal disease * Known active gastrointestinal ulcer * Other severe or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation. * Known diagnosis of myasthenia gravis US only: * Signs or symptoms of myasthenia gravis or stroke during screening * Patients with myasthenia gravis specific autoantibodies or any known history of myasthenia gravis (MG) autoantibodies at screening window * Concomitant medications with the potential to cause de novo myasthenia gravis, worsening of myasthenia gravis or cause myasthenia gravis-like symptoms * Uncontrolled hypertension, atrial fibrillation or flutter, ventricular arrhythmia or receiving treatment for cardiac rhythm disorder or diabetes that is not adequately controlled Other protocol specific inclusion/exclusion criteria may apply

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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

Locations

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan, MI, 20162, Italy

  • ASST Papa Giovanni XXIII

    Bergamo, BG, 24127, Italy

  • ASST Spedali Civili di Brescia

    Brescia, 25123, Italy

  • Azienda Ospedaliero-Universitaria di Bologna - Policlinico S.Orsola-Malpighi

    Bologna, 40138, Italy

  • CHU Hopitaux de Bordeaux - Hôpital Haut-Lévêque

    Pessac, 33604, France

  • Centre Hospitalier Lyon-Sud

    Pierre-Bénite, 69495, France

  • Centre Hospitalier Universitaire de Nantes (CHU de Nantes) - Hotel-Dieu

    Nantes, 44000, France

  • Centre Hospitalier du Mans

    Le Mans, 72037, France

  • Fondazione Policlinico Universitario Agostino Gemelli

    Roma, 00168, Italy

  • Hospital Universitari i Politècnic La Fe

    Valencia, 46026, Spain

  • Istituto Clinico Humanitas

    Rozzano, MI, 20089, Italy

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