BCC018

Eligibility Criteria 

1. Diagnosis: Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. Subjects with the following disease stages at diagnosis are eligible, if they meet the other specified criteria:

2. Subjects with newly diagnosed neuroblastoma with INRGSS Stage M disease with either of the following features:

   1. MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
   2. 365 days to ≥ 547 days of age without MYCN amplification, but unfavorable biologic features such as unfavorable histology (INPC) or diploid tumor (DNA index=1) or the presence of any segmental chromosome aberration (SCA) (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q); OR
   3. Age > 547 days of age regardless of biologic features

   Subjects with newly diagnosed neuroblastoma with INRGSS Stage MS disease with either of the following:

   1. MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals); OR
   2. 365 days to ≥ 547 days (18 months) of age without MYCN amplification, but unfavorable biologic features such as unfavorable histology (INPC) or diploid tumor (DNA index=1) or SCA as above

   Subjects with newly diagnosed neuroblastoma INRGSS Stage L2 disease with either of the following:

   1. MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals); OR
   2. 18 months to <5 years of age without MYCN amplification, but with unfavorable histology (INPC); OR
   3. ≥5 years of age without MYCN amplification, but with undifferentiated or poorly differentiated INPC Subjects with newly diagnosed neuroblastoma INRGSS Stage L1 disease that is incompletely resected with MYCN amplification.

   Subjects > 547 days of age initially diagnosed with INRGSS Stage L1, L2 or MS disease who progressed to Stage M without prior chemotherapy may enroll within 4 weeks of progression to Stage M.

   Subjects ≥ 365 days of age initially diagnosed with MYCN amplified INRGSS Stage L1 disease who progress to Stage M without systemic therapy may enroll within 4 weeks of progression to Stage M.

3. Subjects must be age ≤ 21 years at initial diagnosis.
4. Subjects must be >12 months of age at enrollment.

Please contact Legacy's Cancer and Blood Disorders at Randall Children's Hospital at Legacy Emanuel for additional study inclusion/exclusion information.