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

NCT ID NCT02091245

Ongoing Disease control Sponsor: Dana-Farber Cancer Institute Source: ClinicalTrials.gov ↗

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.

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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.

CHRONIC MYELOGENOUS LEUKEMIA (CML) IN BLAST CRISIS REFRACTORY ACUTE LYMPHOBLASTIC LEUKEMIA (ALL) REFRACTORY ACUTE MYELOGENOUS LEUKEMIA (AML) REFRACTORY BIPHENOTYPIC LEUKEMIA REFRACTORY MIXED LINEAGE LEUKEMIA RELAPSED ACUTE LYMPHOBLASTIC LEUKEMIA (ALL) RELAPSED ACUTE MYELOGENOUS LEUKEMIA (AML) RELAPSED BIPHENOTYPIC LEUKEMIA RELAPSED MIXED LINEAGE LEUKEMIA

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

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