Experimental drug DFMO offered to kids with rare cancers

NCT ID NCT03581240

AVAILABLE Disease control Sponsor: Giselle Sholler Source: ClinicalTrials.gov ↗

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

Summary

This expanded access program provides the drug DFMO to children and young adults up to age 30 with rare relapsed tumors such as neuroblastoma or medulloblastoma. The goal is to control the disease by targeting specific genetic markers. Participants take DFMO by mouth twice daily, with higher doses for those with brain involvement.

What this could mean

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

Active substance: eflornithine (DFMO)
What this could lead to: If successful, this could provide a treatment option for children with rare, hard-to-treat tumors that have come back.
What could go wrong: This is an expanded access study, not a formal trial, so results are limited. DFMO may not work for all tumor types and can cause side effects like hearing loss or stomach issues.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

atypical teratoid rhabdoid tumor ependymoblastoma medulloblastoma medulloepithelioma neuroblastoma Neuroectodermal Tumors, Primitive Typical Teratoid Rhabdoid Tumor

As listed by the trial registrant

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

EMBRYONAL TUMOR WITH ABUNDANT NEUROPIL AND TRUE ROSETTES EPENDYMOBLASTOMA MEDULLOBLASTOMA MEDULLOEPITHELIOMA NEUROBLASTOMA TYPICAL TERATOID RHABDOID TUMOR

Contacts and locations

Locations

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

Hershey, Pennsylvania, 17033, United States

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