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Recruiting Phase 1 NCT06399640

NCT06399640 Eltanexor and Venetoclax in Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia

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Clinical Trial Summary
NCT ID NCT06399640
Status Recruiting
Phase Phase 1
Sponsor Vanderbilt-Ingram Cancer Center
Condition Relapsed Myelodysplastic Syndrome
Study Type INTERVENTIONAL
Enrollment 60 participants
Start Date 2024-08-14
Primary Completion 2027-02-01

Eligibility & Interventions

Sex All sexes
Min Age 18 Years
Max Age N/A
Study Type INTERVENTIONAL
Interventions
EltanexorVenetoclaxBone Marrow Aspiration and Biopsy

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

What to Expect as a Participant

You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.

Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.

This trial targets 60 participants in total. It began in 2024-08-14 with a primary completion date of 2027-02-01.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief Summary

This phase I trial tests the safety, side effects, and best dose of eltanexor in combination with venetoclax for the treatment of patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Eltanexor works by trapping "tumor suppressing proteins" within the cell, thus causing the cancer cells to die or stop growing. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving eltanexor together with venetoclax may be safe, tolerable and/or effective in treating patients with relapsed or refractory MDS or AML.

Eligibility Criteria

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.

Contact & Investigator

Central Contact

Vanderbilt-Ingram Services for Timely Access

✉ cip@vumc.org

📞 800-811-8480

Principal Investigator

Somedeb Ball, MD

PRINCIPAL INVESTIGATOR

Vanderbilt University/Ingram Cancer Center

Frequently Asked Questions

Who can join the NCT06399640 clinical trial?

This trial is open to participants of all sexes, aged 18 Years or older, studying Relapsed Myelodysplastic Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

What phase is the NCT06399640 trial and what does that mean for participants?

Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.

Is NCT06399640 currently recruiting?

Yes, NCT06399640 is actively recruiting participants. Contact the research team at cip@vumc.org for enrollment information.

Where is the NCT06399640 trial being conducted?

This trial is being conducted at Memphis, United States, Nashville, United States.

Who is sponsoring the NCT06399640 clinical trial?

NCT06399640 is sponsored by Vanderbilt-Ingram Cancer Center. The principal investigator is Somedeb Ball, MD at Vanderbilt University/Ingram Cancer Center. The trial plans to enroll 60 participants.

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