New hope for kids with rare cancers: expanded access to DFMO drug

NCT ID NCT03581240

First seen Feb 22, 2026 · Last updated May 14, 2026 · Updated 9 times

Summary

This study offers expanded access to the drug DFMO for children and young adults (ages 0-30) with rare, relapsed tumors like neuroblastoma and medulloblastoma. The drug targets tumors with specific genetic markers (LIN28, MYCN, or ODC). The goal is to provide treatment when no other options are available, aiming to control the disease.

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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: •••••@•••••

Locations

  • Penn State Milton S. Hershey Medical Center and Children's Hospital

    AVAILABLE

    Hershey, Pennsylvania, 17033, United States

    Contact Email: •••••@•••••

    Contact

Conditions

Explore the condition pages connected to this study.