Experimental drug DFMO offered to kids with rare cancers
NCT ID NCT03581240
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.
Disclaimer
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This is a summary of
the original study
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Penn State Milton S. Hershey Medical Center and Children's Hospital
AVAILABLEHershey, Pennsylvania, 17033, United States
Contact Email: •••••@•••••
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