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An Intermediate Expanded Use Trial of DFMO

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.

An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.

Searching Both is inclusive of interventional and observational studies.

Expanded Access
Eligible Ages N/A - 30 Years
Gender All
More Inclusion & Exclusion Criteria

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.

Trial Details

Trial ID:

This trial id was obtained from, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.


Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Giselle SaulnierSholler
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Giselle Sholler, MD
Principal Investigator Affiliation Beat Childhood Cancer
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Overall Status Available
Countries United States

The disease, disorder, syndrome, illness, or injury that is being studied.

Neuroblastoma, Medulloblastoma, Typical Teratoid Rhabdoid Tumor, Embryonal Tumor With Abundant Neuropil and True Rosettes, Ependymoblastoma, Medulloepithelioma
Study Website: View Trial Website

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Levine Children's Hospital, Charlotte, North Carolina




Levine Children's Hospital

Charlotte, North Carolina, 28204

Site Contact

Abigail Moore