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Promising leukemia drug falls short in kids trial

NCT ID NCT03913559

First seen Feb 05, 2026 · Last updated Jun 22, 2026 · Updated 13 times

Summary

This phase 2 trial tested inotuzumab ozogamicin (Besponsa) in children with B-cell acute lymphoblastic leukemia who had low levels of remaining cancer cells (MRD) despite treatment. The drug targets CD22, a protein on leukemia cells, and delivers a potent toxin. Only 5 children were enrolled before the study was terminated early, limiting what can be learned about its effectiveness and safety.

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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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Contacts and locations

Locations

  • Rady Children's Hospital San Diego

    San Diego, California, 92123, United States

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

What this could mean

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

Active substance

Inotuzumab ozogamicin (Besponsa)

What this could lead to

If successful, this could offer a new option to clear remaining leukemia cells in children with high-risk B-ALL, potentially improving outcomes before or after transplant.

What could go wrong

The trial was terminated early with only 5 participants, so results are very limited. The drug carries a risk of liver damage and veno-occlusive disease, especially after transplant.

Conditions

The condition(s) this trial relates to.

Burkitt lymphoma Precursor Cell Lymphoblastic Leukemia-Lymphoma

As listed by the trial registrant

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