New drug combo aims to outsmart resistant leukemia
NCT ID NCT06399640
First seen Jun 26, 2026 · Last updated Aug 20, 2026 · Updated 3 times
Summary
This early-phase trial is testing whether combining two oral drugs, eltanexor and venetoclax, is safe and effective for people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has returned or not responded to prior treatment. The study will enroll 60 adults and look for the best dose and any signs of remission. Both drugs work by blocking proteins that help cancer cells survive.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- eltanexor and venetoclax
- What this could lead to
- If this works, it could offer a new treatment option for people with blood cancers that have stopped responding to standard therapies.
- What could go wrong
- This is an early Phase 1 trial with only 60 participants, so it is primarily testing safety and dosing. It may not lead to a proven treatment, 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 60 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2024
- Expected to finish
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Oct 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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: \- Age \>/= 18 years at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and must be able to meet all study requirements. For Myelodysplastic Syndrome (MDS): Morphologically confirmed diagnosis of MDS with increased blasts (\>/= 5%), with a prior DNA methyltransferase inhibitor (DNMTi) treatment and progression after 2 cycles or stable disease after 4 cycles For Acute Myeloid Leukemia (AML): Morphologically confirmed diagnosis of AML in accordance with WHO diagnostic criteria that is relapsed or refractory following \>/= 1 line(s) of therapy. * WBC must be less than 25,000/ul prior to study start (hydroxyurea allowed). * A bone marrow aspirate must be performed, and tissue collected for entrance to the trial unless circulating blasts \>/= 5% in which case, peripheral blood can be used. * Eastern Cooperative Oncology Group Performance Status of 0 - 2. * Must have adequate hepatic and renal function as demonstrated by the following: ALT(SGPT) and/or AST (SGOT) \</= 3x upper limit of normal (ULN); Total bilirubin \</= 1.5x ULN; Calculated creatinine clearance \> 50 ml/min (per the Cockroft-Gault formula). \- Willingness to abide by all study requirements, including contraception, maintenance of a pill diary, and acceptance of recommended supportive care medications. Exclusion Criteria: * Anticancer therapy, including investigational agents \</= 2 weeks or \</= 5 half-lives of the drug, whichever is shorter, prior to C1D1. (Use of hydroxyurea is permitted). * Inadequate recovery from toxicity attributed to prior anti-cancer therapy to \</= Grade 1 (NCI CTCAE v5.0), excluding alopecia or fatigue. * Prior treatment with SINE compounds or other inhibitors of XPO1. * History of allogeneic hematopoietic stem cell transplant (HCT), or other cellular therapy product, within 3 months. * Active acute or chronic GVHD requiring calcineurin inhibitors or steroid dosing \>/= 10mg/day or patients within 4 weeks of stopping calcineurin inhibitors for GVHD. * Radiation therapy or major surgery within 3 weeks. * Active, uncontrolled infection. Patients with infection under active treatment and controlled with antibiotics are eligible. Prophylaxis, even if parenteral, is acceptable. * Inability to swallow oral medications. * Active documented central nervous system leukemia. * Second active malignancy within past 2 years except for basal or squamous cell carcinoma of the skin, ductal carcinoma of breast in situ or cervical carcinoma in situ. * Women of childbearing age or potential must have negative pregnancy test and must not be actively breastfeeding to enroll on the study * Clinically significant cardiovascular disease with major event or cardiac intervention within the past 6 months (e.g. percutaneous intervention, coronary artery bypass graft, documented cardiac heart failure) as determined by the investigator. * QT interval corrected by Fridericia's formula (QTcF)\>470msec for both males and females on screening ECG. Patients with a bundle branch block or in-situ pacemaker must have appropriate QT interval corrections for these conditions. * Any condition not listed but deemed by the investigator to make the patient a poor candidate for clinical trial and/or treatment with investigational agents.
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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
2 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
Locations
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Baptist Health Care Corporation
RECRUITINGMemphis, Tennessee, 38138, United States
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Vanderbilt University/Ingram Cancer Center
RECRUITINGNashville, Tennessee, 37203, United States
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Other studies related to the condition(s) this trial covers.
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- Two-Drug combo targets leukemia that outsmarted its first treatment
- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?
- New drug combination targets Hard-to-Treat blood cancers