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.
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).
High risk neuroblastoma patients that have completed standard of care upfront therapy and are not eligible for NMTRC014.
Medulloblastoma patients who have completed standard of care therapies.
BSA (m2) of <0.25
Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
Giselle Sholler
Lead sponsor
Milton S. Hershey Medical Center
Sponsor institution
K C Pharmaceuticals Inc.
Collaborator
USWM, LLC
Collaborator