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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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0 to 30 years
- Sex
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Anyone
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Age: 0-30 years at the time of initial diagnosis. * Diagnosis: Histologic verification at either the time of original diagnosis or a previous relapse of High Risk neuroblastoma, medulloblastoma, atypical teratoid rhabdoid tumor, embryonal tumor with abundant neuropil and true rosettes, ependymoblastoma, medulloepithelioma and other rare pediatric MYC, ODC or LIN28/Let7 driven tumors (each type will form a new subset). * Disease Status: Subjects must be in one of the following disease categories: 1. High risk neuroblastoma patients that have completed standard of care upfront therapy and are not eligible for NMTRC014. 2. Medulloblastoma patients who have completed standard of care therapies. 3. Relapsed/refractory neuroblastoma patients who have completed standard of care therapies. 4. Rare tumors with increased LIN28 expression or MYCN amplification or up regulation of ornithine decarboxylase who have completed standard of care therapies. * Subjects are not eligible to enroll on DFMO studies NMTRC002, NMTRC003, NMTRC010, or NMTRC014. * A negative serum or urine pregnancy test is required for female subjects of child bearing potential (onset of menses or ≥13 years of age). * Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended. * Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines Exclusion Criteria: * BSA (m2) of \<0.25 * Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Penn State Milton S. Hershey Medical Center and Children's Hospital
AVAILABLEHershey, Pennsylvania, 17033, United States
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