Testing Naxitamab for neuroblastoma

Inclusion

• +Subjects must be age ≤ 21 years at initial diagnosis.
• +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:
• +Subjects with newly diagnosed neuroblastoma with INRGSS Stage M disease with either of the following features:
• +MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
• +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
• +Age \> 547 days of age regardless of biologic features
• +Subjects with newly diagnosed neuroblastoma with INRGSS Stage MS disease with either of the following:
• +MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals); OR
• +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:
• +MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals); OR
• +18 months to \<5 years of age without MYCN amplification, but with unfavorable histology (INPC); OR
• +≥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 ≥ 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.
• +Subjects must be \>12 months of age at enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →.
• +Adequate cardiac function defined as:
• +Shortening fraction of ≥ 27% by echocardiogram, or
• +Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.
• +Adequate liver function must be demonstrated, defined as:
• +Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
• +ALT (SGPT) \< 5 x upper limit of normal (ULN) for age
• +1\. Subjects must have adequate renal function defined as:
• +For subjects \< 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside Schwartz equation is: \[(0.413) X (Height in cm)\] / SCr
• +For subjects ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: \[(140-age) x (Wt in kg) x (0.85 if female)\] / (72 x SCr)
• +OR a 24 hour urine Creatinine clearance ≥ 70 mL/min/1.73 m2
• +Informed ConsentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines and applicable local regulations.
• +A negative serum pregnancy test is required for subjects of childbearing potential (≥13 years of age or after onset of menses)
• +Both male and female post-pubertal study subjects must be willing to use a highly effective contraceptive method (i.e., achieves a failure rate of \<1% per year when used consistently and correctly) from the time of informed consent (and assent, as applicable) until 6 months after study treatment discontinuation. Such methods include: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner, sexual abstinence.
• +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.