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New pill shows promise for tough blood cancers

NCT ID NCT05732103

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests an experimental drug called CTX-712 in people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have come back or not responded to treatment. The drug is taken by mouth once or twice weekly. The trial aims to find the safest dose and see if it can shrink or control the cancer. About 225 adults will take part across multiple centers.

What this could mean

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

Active substance
CTX-712 (an experimental oral drug taken once or twice weekly)
What this could lead to
If it works, this could offer a new treatment option for people with hard-to-treat AML or MDS, potentially helping control the disease.
What could go wrong
This is an early-phase trial (Phase 1/2) with only 225 participants, so the drug may not work or could have serious side effects. It is not a cure and long-term benefits 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

Phase 1/2

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

Participants

About 225 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2023

Expected to finish

Feb 2029

An estimate. End dates often move.

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: 1. Age ≥18 years. 2. Diagnosis of AML, HR-MDS, or high marrow blast MDS/MPN (including CMML). Note: Only patients with AML and HR-MDS are eligible in the expansion cohorts. 3. Prior treatment history must include 1-4 prior lines of therapy. Note: 1-3 prior lines are allowed for patients in the expansion cohorts. 4. Adequate organ function evidenced by the following laboratory values: Creatinine clearance (CL) ≥60 mL/min * Total serum bilirubin \< 1.5 × upper limit of normal (ULN) * Alanine aminotransferase (ALT) * Aspartate aminotransferase(AST) \< 2.5 × ULN * White blood cell count at the time of the first dose \<10 k/μL 5. Eastern Cooperative Oncology Group performance status ≤2. 6. Female patients of childbearing potential must have a negative pregnancy test within 7 days before study treatment initiation and if sexually active, agree to use a highly effective form of contraception throughout their participation during study treatment and up to 4 months after the last dose of study drug. 7. Male patients with female partners of childbearing potential must, even if surgically sterilized, agree to practice effective barrier contraception during the entire study treatment period and through four months after the last dose of study drug, or practice true abstinence, when this is in line with the preferred and usual lifestyle of the participant. Exclusion criteria: 1. Diagnosis of acute promyelocytic leukemia. 2. Isolated extramedullary relapse (phase 2 only). 3. Active central nervous system (CNS) leukemia. 4. History of other malignancy. 5. Any of the following cardiopulmonary abnormalities: 1. Myocardial infarction within six months prior to registration. 2. New York Heart Association Class III or IV heart failure or known left ventricular ejection fraction \< 50%. 3. A history of familial long QT syndrome. 4. Symptomatic atrial or ventricular arrhythmias not controlled by medications. 5. QTcF ≥ 470 msec calculated according to institutional guidelines, unless due to underlying bundle branch block and/or pacemaker and with approval of the medical monitor. 6. Known moderate to severe and clinically significant chronic obstructive pulmonary disease, interstitial lung disease and/or pulmonary fibrosis (e.g., requiring home oxygen therapy). 6. Pregnancy and/or lactation. 7. Major surgery (excluding placement of vascular access) within 4 weeks prior to first dose of CTX-712. 8. History of allogeneic organ transplantation (excluding cornea). 9. History of allogenic hematopoietic stem cell transplantation within 6 months of planned study treatment initiation and/or graft-versus host disease grade ≥ 1 following allogenic hematopoietic stem cell transplantation. 10. History of or chimeric antigen receptor T-cell therapy or other modified T cell therapy. 11. Active, uncontrolled bacterial, fungal, or viral infection, including hepatitis B virus, hepatitis C virus, known human immunodeficiency virus, or acquired immunodeficiency syndrome related illness. Infections controlled with oral anti-infective agents, including prophylactic treatments, are allowed. Patient must be viral load negative. 12. Psychological, familial, sociological, or geographical conditions that do not permit compliance with the protocol and/or follow-up procedures outlined in the protocol.

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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.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    8 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. 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

  • Fred Hutchinson Cancer Center

    RECRUITING

    Seattle, Washington, 98109, United States

  • Mayo Clinic Arizona

    RECRUITING

    Phoenix, Arizona, 85054, United States

  • Mayo Clinic Comprehensive Cancer Center

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Mayo Clinic Florida

    RECRUITING

    Jacksonville, Florida, 32224, United States

  • Northwestern University

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact Email: •••••@•••••

  • The University of Rochester

    RECRUITING

    Rochester, New York, 14642, United States

    Contact Email: •••••@•••••

  • The University of Texas MD Anderson Cancer Center

    RECRUITING

    Houston, Texas, 77030, United States

  • University of Virginia

    RECRUITING

    Charlottesville, Virginia, 22903, United States

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