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Eltanexor and Venetoclax for Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia

This study is testing the safety, side effects, and best dose of two oral medications, eltanexor and venetoclax, for people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). These are types of blood cancers that have either returned after treatment (relapsed) or have not responded to previous treatments (refractory). Eltanexor works by helping cancer cells die, while venetoclax blocks a protein that cancer cells need to survive. Researchers want to see if giving these two drugs together is safe and effective. You must be at least 18 years old and have a confirmed diagnosis of MDS with increased blasts or AML that has relapsed or is refractory to join. The study aims to enroll 60 participants.

Study design
This is a dose-escalation study that will enroll 60 participants. It is testing two medications given together.
What's involved
You would take eltanexor and venetoclax by mouth daily for specific periods within each 28-day cycle. You will also have bone marrow aspirations and biopsies, and blood samples collected throughout the study.
Compensation
Not stated in the trial record.
Follow-up
The study will track adverse events and the biologically effective dose for up to 2 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06399640

Eltanexor and Venetoclax in Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia

Recruiting
PHASE1Ages 18+InterventionalTreatment
Vanderbilt-Ingram Cancer Center
~60 participants
Updated 2026-08-19 on ClinicalTrials.gov
What's tested:EltanexorVenetoclaxBone Marrow Aspiration and BiopsyBiospecimen Collection

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of adverse events
Measured over Up to 2 years
+1 more outcome measured

Conditions

Relapsed Myelodysplastic Syndrome
Refractory Myelodysplastic Syndrome
Acute Myeloid Leukemia
Recurrent Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia

Explore related trials

Acute Myeloid LeukemiaMyelodysplastic Syndrome

Where it's being run

2 sites across 1 states
Tennessee2

Study leadership

  • Somedeb Ball, MD · PRINCIPAL_INVESTIGATOR · Vanderbilt University/Ingram Cancer Center

Who to contact

Vanderbilt-Ingram Services for Timely Access
[email protected]
800-811-8480
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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:

Exclusion

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.

What this trial measures

  • Incidence of adverse eventsUp to 2 years

    Adverse medical events will be tabulated and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5.

  • Biologically effective dose (BED) of eltanexor in combination with venetoclaxUp to 2 years

    Measured by complete remission