An Intermediate Expanded Use Trial of DFMO

NCT03581240•Giselle Sholler

Summary

To provide DFMO in an expanded use setting to subjects with relapsed rare tumors with increased LIN28 expression or MYCN amplification or up regulation of ornithine decarboxylase.

Eligibility

Age

0 - 30 years

Sex

ALL

Healthy Volunteers

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:

Exclusion Criteria

•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.
•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
•BSA (m2) of \<0.25
•Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.

Locations (1)

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

Hershey, Pennsylvania, United States

Key Dates

Last Updated

February 20, 2026

Conditions

NeuroblastomaMedulloblastomaTypical Teratoid Rhabdoid TumorEmbryonal Tumor With Abundant Neuropil and True RosettesEpendymoblastomaMedulloepithelioma

Interventions

eflornithine HCl

DRUG

Contacts

BCC Enroll

CONTACT

7175310003 BCCEnroll@pennstatehealth.psu.edu

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 20, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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An Intermediate Expanded Use Trial of DFMO

Inclusion Criteria

Exclusion Criteria

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