- Diagnosis: Subjects must have a diagnosis of high-risk (defined in protocol)
neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology
or demonstration of clumps of tumor cells in bone marrow with elevated urinary
- 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.
- Adequate liver function must be demonstrated (defined in protocol)
- Subjects must have adequate renal function defined as a Serum creatinine based on
age/gender (defined in protocol)
- Adequate Cardiac Function (defined in protocol)
- Ability to tolerate PBSC collection: No known contraindication to PBSC collection.
- A negative serum pregnancy test is required for female participants of child bearing
potential (13 years of age or after onset of menses)
- 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
- 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
- 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
Minimum Eligible Age: 12 Months
Maximum Eligible Age: 21 Years
Eligible Gender: Both
Number of subjects that have a targeted agent chosen for treatment.
Number of subjects that receive 75% of dosing of medications while on study protocol during cycles 3-6.
Number of subjects required to go off therapy due to treatment-related adverse events as assessed by CTCAE v4.0.
Number of subjects that are able to complete 2 years of DFMO therapy.