Actively Recruiting

Phase 1
Age: 18Years +
All Genders
NCT06399640

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

Led by Vanderbilt-Ingram Cancer Center · Updated on 2025-03-27

60

Participants Needed

1

Research Sites

163 weeks

Total Duration

On this page

Sponsors

V

Vanderbilt-Ingram Cancer Center

Lead Sponsor

K

Karyopharm Therapeutics Inc

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age �3 18 years at the time of signing the Informed Consent Form (ICF)
  • Must voluntarily sign an ICF and meet all study requirements
  • For MDS: Diagnosis confirmed with increased blasts (�3 5%) and prior DNA methyltransferase inhibitor treatment with progression or stable disease
  • For AML: Diagnosis confirmed according to WHO criteria with relapsed or refractory disease after �3 1 prior therapy
  • White blood cell count less than 25,000/ul before starting study (hydroxyurea allowed)
  • Bone marrow aspirate performed unless circulating blasts �3 5%, then peripheral blood can be used
  • Eastern Cooperative Oncology Group Performance Status of 0 to 2
  • Adequate liver and kidney function with specified lab values
  • Willingness to follow all study requirements including contraception and pill diary
Not Eligible

You will not qualify if you...

  • Anticancer therapy including investigational agents within 2 weeks or 5 half-lives prior to first treatment day (except hydroxyurea)
  • Incomplete recovery from prior therapy side effects to �3 Grade 1 (except alopecia or fatigue)
  • Prior treatment with SINE compounds or XPO1 inhibitors
  • History of allogeneic stem cell transplant or cellular therapy within 3 months
  • Active graft-versus-host disease requiring certain medications or recent stopping of those medications
  • Radiation therapy or major surgery within 3 weeks
  • Active uncontrolled infection (controlled infections with antibiotics allowed)
  • Unable to swallow oral medications
  • Active central nervous system leukemia
  • Second active cancer within 2 years except certain skin and in situ cancers
  • Women of childbearing potential must have negative pregnancy test and not be breastfeeding
  • Significant cardiovascular disease or recent major cardiac events
  • Any condition judged by investigator as making patient a poor candidate for trial or treatment

AI-Screening

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Trial Site Locations

Total: 1 location

1

Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, United States, 37203

Actively Recruiting

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Research Team

V

Vanderbilt-Ingram Services for Timely Access

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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