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New hope for tough blood cancers: venetoclax combo enters human trials

NCT ID NCT05292664

First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 38 times

Summary

This early-phase trial is testing whether adding the drug venetoclax to standard chemotherapy is safe and tolerable for children, adolescents, and young adults with high-risk blood cancers like MDS, AML, and ALL. About 30 participants will receive different combinations of chemotherapy drugs along with venetoclax. The main goal is to find the best dose and watch for side effects, not yet to prove the treatment works.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Ann & Robert H Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Healthcare of Atlanta at Arthur M. Blank Hospital

    RECRUITING

    Atlanta, Georgia, 30329, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Dana-Farber Cancer Institute

    RECRUITING

    Boston, Massachusetts, 02215, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of California San Francisco-Benioff Children's Hospital

    RECRUITING

    San Francisco, California, 94158, United States

    Contact Email: •••••@•••••

What this could mean

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

Active substance

Venetoclax (Venclexta) combined with chemotherapy drugs including azacitidine, cytarabine, methotrexate, and calaspargase pegol

What this could lead to

If it works, this could lead to a safer, more effective treatment option for children and young adults with hard-to-treat blood cancers.

What could go wrong

This is a very early (Phase 1) trial with only 30 participants, focused on safety and dosing. The combination may cause serious side effects, and it's too soon to know if it will improve outcomes.

Conditions

The condition(s) this trial relates to.

acute biphenotypic leukemia acute lymphoblastic leukemia Myelodysplastic Syndromes Neoplasm Metastasis Precursor Cell Lymphoblastic Leukemia-Lymphoma Recurrence

As listed by the trial registrant

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