Next Generation Personalized Neuroblastoma Therapy

Inclusion Criteria:

- Aged ≥1 years to ≤ 21 years

- Relapsed or refractory neuroblastoma

- A sufficient interval between the last dose of prior anti-cancer therapy (including
cytotoxic and biological therapies) and enrollment in this study, to allow recovery
from the acute toxic effects of all prior anti-cancer therapy. Please contact site for
specific details

- Adequate bone marrow function (bone marrow may be involved with tumor. Contact site
for specific details)

- Adequate renal function, defined as Creatinine clearance or radioisotope glomerular
filtration rate (GFR) ≥ 70 mL/min/1.73 m2 OR serum creatinine based on age/gender
normal (contact site for details)

- Adequate liver function, defined as total serum bilirubin ≤ 1.5 times the upper limit
of normal AND alanine transaminase (ALT) ≤ 110 U/L.

- Adequate cardiac function, defined as corrected QT interval (QTc) ≤ 480 msec AND
shortening fraction > 27%

- Males and females who are sexually active must agree to use effective contraception
during and for 3 months after treatment

Exclusion Criteria:

- Subjects taking certain drugs or herbal medications that impact drug metabolism and/or
cardiac function that cannot be discontinued (contact site for details).

- Subjects with concurrent severe and/or uncontrolled concurrent medical conditions that
could compromise participation in the study (contact site for details)

- Other concomitant therapies:

- Corticosteroids initiated for tumor therapy within 7 days prior to study enrollment

- Other anti-cancer agents

- Other investigational drugs

- Hematological growth factors

- Radiation therapy

- Subjects < 0.5m2

- Pregnant or lactating females

- Sexually active males unless they use a condom during intercourse while taking study
drug/s and for 3 months after study drug discontinuation and thus do not attempt to
father a child in this period.