New drug selinexor tested in kids with Tough-to-Treat leukemia

NCT ID NCT02091245

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial is testing the drug selinexor (KPT-330) in 16 children and young adults with leukemia that has come back or not responded to standard treatments. The main goal is to find the safest dose and understand side effects. The drug works by blocking a protein that leukemia cells need to survive, potentially killing them.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Selinexor (KPT-330)

What this could lead to

If it works, this could point toward a new treatment option for children with leukemia that has not responded to standard therapies.

What could go wrong

This is a very early phase 1 trial with only 16 participants, so it is primarily testing safety and dosing. The drug may not effectively control the leukemia, and side effects could be significant.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

acute biphenotypic leukemia acute leukemia of ambiguous lineage acute lymphoblastic leukemia acute myeloid leukemia blast phase chronic myelogenous leukemia, BCR-ABL1 positive Leukemia, Myelogenous, Chronic, BCR-ABL Positive mixed phenotype acute leukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98101, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • UCSF

    San Francisco, California, 94143, United States