Preventative Trial of DFMO in Patients With High Risk Neuroblastoma in Remission
Preventative Trial of DFMO in Patients With High Risk Neuroblastoma in Remission
The purpose of this research study is to evaluate a new investigational drug to prevent
 reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The
 objectives of this study will be to monitor for safety and look at efficacy of DFMO.

 The safety of the proposed dosing regimen in this trial will be tested by an on-going
 risk/benefit assessment during the study. A patient benefiting from treatment, not
 progressing on therapy, and in the absence of any safety issues associated with DFMO may
 continue on treatment up to 27 cycles with the expectation that there will be an overall
 clinical benefit.

 The procedures involved in this study include Medical history, Physical exam, Vital signs
 (blood pressure, pulse, temperature), Blood tests, Urine tests, MRI or CT scan of the
 tumor(s), MIBG scans, and Bone marrow aspirations. All of these tests and procedures are
 considered standard of care for this population. Drug administration is also part of this
 protocol, including an investigational new drug called DFMO.

 The proposed dosing regimen is an oral dose of DFMO tablets two times a day for each day
 while on study. There will be 27 cycles. Each cycle will be 28 days in length.
Inclusion Criteria:

 -  Age: 0-21 years at the time of diagnosis.

 -  Diagnosis: histologic verification at either the time of original diagnosis or a
 previous relapse of high risk neuroblastoma.

 -  Disease Status: Neuroblastoma that is in remission

 -  Greater than 30 days from completion of cytotoxic and biologic therapy and less than
 120 days from previous therapy.

 -  A negative urine pregnancy test is required for female subjects of child bearing
 potential (onset of menses or ≥13 years of age).

 -  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.

 -  ANC > 500/μl and platelet count >50,000/μl

 -  Organ Function Requirements: Subjects must have adequate liver function as defined by:

 -  AST and ALT <10x upper limit of normal

 -  Serum bilirubin must be ≤ 2.0 mg/dl

 -  Serum creatinine based on age/gender

 -  Informed Consent: All subjects and/or legal guardians must sign informed written
 consent. Assent, when appropriate, will be obtained according to institutional
 guidelines

 Exclusion Criteria:

 -  Lansky score < 60%

 -  BSA (m2) of <0.25

 -  Investigational Drugs: Subjects who are currently receiving another investigational
 drug are excluded from participation.

 -  Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are
 not eligible. Subjects must have fully recovered from the effects of prior
 chemotherapy (hematological and bone marrow suppression effects).

 -  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.

 -  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.