New hope for kids with tough cancers: ponatinib trial launches

NCT ID NCT03934372

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests the drug ponatinib in children aged 1 to 18 with advanced leukemias, lymphomas, or solid tumors that have come back or not responded to standard treatment. The goal is to find safe doses and see if the drug can shrink tumors or improve blood counts. About 70 children will take part across multiple hospitals.

What this could mean

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

Active substance

ponatinib (a targeted cancer drug)

What this could lead to

If it works, this could provide a new treatment option for children with hard-to-treat cancers that have not responded to other therapies.

What could go wrong

This is an early-phase trial with only 70 participants, so results may not apply to all children. The drug can cause serious side effects like blood clots and liver problems.

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 lymphoblastic leukemia acute myeloid leukemia blast phase chronic myelogenous leukemia, BCR-ABL1 positive chronic myelogenous leukemia, BCR-ABL1 positive leukemia leukemia, myeloid, accelerated-phase Leukemia, Myeloid, Chronic-Phase lymphoma neoplasm 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

Study contacts

  • Contact

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

Locations

  • A.O.U Citta Della Salute E Della Scienza Di Torino Presidio Ospedaliero Infantile Regina Margherita

    RECRUITING

    Torino, 10126, Italy

  • Alder Hey Childrens Nhs Foundation Trust

    RECRUITING

    Liverpool, L12 2AP, United Kingdom

  • Aorn Santobono Pausilipon

    NOT_YET_RECRUITING

    Naples, 80122, Italy

  • Aou Policlinico S. Orsola-Malpighi

    RECRUITING

    Bologna, 40138, Italy

  • Armand Trousseau Hospital

    RECRUITING

    Paris, 75571, France

  • Asst Degli Spedali Civili Di Brescia

    RECRUITING

    Brescia, 25123, Italy

  • Centre Hospitalier Universitaire de Poitiers

    RECRUITING

    Poitiers, 86021, France

  • Chu de Rennes - Hospital Sud

    RECRUITING

    Rennes, 35700, France

  • Comitato Di Bioetica Della Fondazione Irccs Policlinico San Matteo

    RECRUITING

    Pavia, 27100, Italy

  • Comitato Etico Fondazione Irccs Istituto Nazionale Dei Tumori Milano

    RECRUITING

    Milan, 20133, Italy

  • Ghent University Hospital

    RECRUITING

    Ghent, 09000, Belgium

  • Hopital Robert Debre

    RECRUITING

    Paris, 75019, France

  • Hospital General Universitario Vall D Hebron

    RECRUITING

    Barcelona, 08035, Spain

  • Hospital Infantil Unversitario Nino Jesus

    RECRUITING

    Madrid, 28009, Spain

  • Hospital Sant Joan de Deu de Manresa

    RECRUITING

    Barcelona, 08035, Spain

  • Hospital Universitari I Politecnic La Fe

    RECRUITING

    Valencia, 46026, Spain

  • Karolinska University Hospital Solna

    RECRUITING

    Stockholm, 14141, Sweden

  • Ospedale Pediatrico Bambino Gesu Irccs

    RECRUITING

    Rome, 00165, Italy

  • Ospedale Pediatrico G. Gaslini

    RECRUITING

    Genova, 16147, Italy

  • Princess Maxima Center For Pediatric Oncology

    RECRUITING

    Utrecht, 03584, Netherlands

  • Royal Hospital For Sick Children Yorkhill Glasgow

    RECRUITING

    Glasgow, G514TF, United Kingdom

  • The Royal Marsden Nhs Foundation Trust - Sutton

    RECRUITING

    Sutton, SM2 5PT, United Kingdom

  • University of Milano Bicocca

    RECRUITING

    Monza, 20900, Italy