New pill shows promise for tough blood cancers
NCT ID NCT05732103
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
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 225 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2023
- Expected to finish
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Feb 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: 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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Genom att skicka in godkänner du våra Användarvillkor
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 study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
8 sites. 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
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
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Fred Hutchinson Cancer Center
RECRUITINGSeattle, Washington, 98109, United States
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Mayo Clinic Arizona
RECRUITINGPhoenix, Arizona, 85054, United States
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Mayo Clinic Comprehensive Cancer Center
RECRUITINGRochester, Minnesota, 55905, United States
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Mayo Clinic Florida
RECRUITINGJacksonville, Florida, 32224, United States
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Northwestern University
RECRUITINGChicago, Illinois, 60611, United States
Contact Email: •••••@•••••
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The University of Rochester
RECRUITINGRochester, New York, 14642, United States
Contact Email: •••••@•••••
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The University of Texas MD Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
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University of Virginia
RECRUITINGCharlottesville, Virginia, 22903, United States
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