A Study of a Vaccine in Combination With β-glucan and GM-CSF in People With Neuroblastoma

Inclusion Criteria:

• Diagnosis of NB as defined by international criteria, i.e., histopathology (confirmed by the MSK Department of Pathology) or BM metastases plus high urinecatecholamine levels or positivity in MIBG scan

• HR-NB as defined by risk-related treatment guidelines and internationalcriteria,i.e., metastatic/non-localized disease with MYCN amplification (any age),MYCN-non-amplified metastatic disease >18 months old, MYCNamplified localizeddisease (any age), or disease resistant to standard chemotherapy.

• HR-NB (as defined above) and in 1) first CR at ≥ 6 months from initiation ofimmunotherapy using anti-GD2 antibody, or 2) second or subsequent CR (achieved aftertreatment for PD). CR is defined according to the International NeuroblastomaResponse Criteria.Patients with positive MIBG scan but negative FDG-PET scan, and CRin BM, are eligible.

• Patients with grade 3 toxicities or less using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA (CTCAE v5.0) related tohematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinalfunction as determined by blood tests or physical exam.

• Hematologic Function

• Absolute neutrophil count (ANC) ≥ 500/mcl

• Absolute lymphocyte count ≥ 500/mcl

• Hemaglobin (Hgb) ≥ 8 g/dL

• Platelet count ≥ 50,000 mm^3

• Renal Function o Serum creatinine ≤ 3.0 x ULN

or

• eGFR >60 mL/min/1.73 m^2

• Hepatic Function

• Serum bilirubin ≤ 3.0 × ULN

• Aspartate transaminase (AST) ≤ 5.0 × ULN

• Alanine aminotransferase (ALT) ≤ 5.0 × ULN

• Prior treatment with other immunotherapy, including mAbs or vaccine, is allowedbut must be completed ≥ 21 days before the 1st vaccination.

Note: Prior treatment with an investigational therapy must be completed ≥ 28 days before the 1st vaccination.

• ≥ 21 and ≤ 180 days between completion of systemic therapy and 1st vaccination.

• Patients have recovered from any toxicities grade 3 or higher caused by priortherapies.

• Patients previously enrolled on this trial are eligible for repeat enrollment ifthey did not complete all vaccine injections during the first time on protocol butthey will be assigned to Group 3 and will not be included in the primarybiostatistical analyses.

• A negative pregnancy test is required for patients w ith child-bearing capability.

• Signed informed consent indicating awareness of the investigational nature of thisprogram.

Exclusion Criteria:

• Patients w ith significant (grade >4) hematologic, cardiac, neurological, pulmonary,renal, hepatic or gastrointestinal function as determined by blood tests or physicalexam, using the Common Toxicity Criteria (Version 5.0) developed by the NationalCancer Institute of the USA (CTCAE v5.0)

• History of allergy to KLH, QS-21, OPT-821, or glucan.

• Active life-threatening infection requiring systemic therapy.

• Inability to comply with protocol requirements.

• Patients with history of allergy to GM-CSF or who are unable to obtain GM-CSFbecause of insurance issues are ineligible