BCC016

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.

   Cohort 1- Molecular High Risk:

   • Metastatic non-MYC amplified Group 3
   • Metastatic Group 4
   • Metastatic non-WNT/non-SHH (Must be non-MYC amplified)

   Cohort 2- Molecular Very High Risk

   • Metastatic OR MYCN amplified OR TP53 mutant non-infant (>3 yrs) SHH
   • MYC amplified Group 3
   • Non-WNT, non-SHH infant (< 3 yrs)

   Cohort 3: Relapsed/Refractory Medulloblastoma

3. Pre-enrollment tumor survey:

   Prior to enrollment on this study, a determination of mandatory disease staging must be performed:

   • 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.

   *Stable residual disease defined as non-progression over 2 separate imaging studies at least 6 weeks apart

   **Non-bulky disease defined as maximal cross-sectional area < 3cm^2 at enrollment. Patients with leptomeningeal disease are allowed to participate on study.

5. Timing from prior therapy:

   Enrollment (first dose of DFMO) no later than 60 days after last dose of conventional chemotherapy. Patients who have undergone high dose chemotherapy (HDCT) with autologous stem cell transplantation (SCT) are eligible if more than 45 days have elapsed since date of last SCT.

Please contact Legacy's Cancer and Blood Disorders at Randall Children's Hospital at Legacy Emanuel for additional study inclusion/exclusion information.