DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

Eligibility Criteria

• 1. Age: 0-21 years of age at diagnosis
• 2. Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling.
• * Metastatic non-MYC amplified Group 3
• * Metastatic Group 4
• * Metastatic non-WNT/non-SHH (Must be non-MYC amplified)
• * Metastatic OR MYCN amplified OR TP53 mutant non-infant (\>3 yrs) SHH
• * MYC amplified Group 3
• * Non-WNT, non-SHH infant (\< 3 yrs)
• 3. Pre-enrollment tumor survey:
• * Tumor imaging studies including: Brain and spine MRI
• * Lumbar Puncture only if previously positive
• * Bone Marrow aspiration/biopsy only if previously positive
• * This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to first dose of study drug, but must be done within a maximum of 4 weeks before first dose of study drug.
• 4. Disease Status: Subjects must have no evidence of disease, or stable\* residual nonbulky\*\* disease.
• 5. Timing from prior therapy:
• 6. Patients must have a Lansky or Karnofsky Performance Scale score of ≥ 50% (see Appendix II) and patients must have a life expectancy of ≥ 2 months.
• 7. All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to first dose of study drug unless otherwise indicated below.
• 8. Patients must have adequate organ functions at the time of registration:
• * Hematological: Hematological recovery as defined by ANC ≥750/μL, platelets ≥30 (non-transfused x 7 days)
• * Liver: Adequate liver function as defined by AST and ALT \<10x upper limit of normal
• * Renal: Adequate renal function defined as (perform one of the following): Creatinine clearance or radioisotope GFR ≥ 70 mL/min/1.73 m2 or a serum creatinine based on age/gender
• 9. Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding.
• 10. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or patients' legal representative).
• 1. BSA of \<0.25 m2
• 2. Metastatic disease outside of CNS
• 3. Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
• 4. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
• 5. Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
• 6. Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
• 7. Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.