About this trial
A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma.
Eligibility criteria
Qualifiers
Subjects must be age ≤ 21 years at initial diagnosis
Subjects must not have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology.
Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.
Ability to tolerate PBSC collection: No known contraindication to PBSC collection. Examples of contraindications would include a weight or size less than that determined to be feasible at the collecting institution, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure.
Disqualifiers
Subjects who are 12-18 months of age with INSS Stage 4 and all stage 3 subjects with favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index > 1) are not eligible.
Lactating females are not eligible unless they have agreed not to breastfeed their infants.
Subjects receiving any investigational drug concurrently.
Subjects with any other medical condition, including but not limited to malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study
Trial design
Treatments tested in this trial
- Ceritinib
- dasatinib
- sorafenib
- vorinostat
- DFMO
Treatment groups
Sponsors and collaborators
Giselle Sholler
Lead sponsor
Milton S. Hershey Medical Center
Sponsor institution
Dell, Inc.
Collaborator
Beat NB Cancer Foundation
Collaborator
K C Pharmaceuticals Inc.
Collaborator
Team Parker for Life
Collaborator