A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma

Study Overview
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Brief Summary
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The purpose of this study is to test which treatment schedule of β-glucan with bivalent vaccine is more effective for participants with high-risk neuroblastoma that is in complete remission.

Official Title
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Phase II Trial of a Bivalent Vaccine With the Immunological Adjuvant OPT-821 (QS-21), in Combination With Randomization of Oral β-glucan, for High-Risk Neuroblastoma

Conditions
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Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma

Intervention / Treatment
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* Biological: OPT-821 (QS-21)
* Dietary Supplement: oral β-glucan


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of NB as defined by international criteria,102 i.e., histopathology (confirmed by the MSK Department of Pathology) or BM metastases plus high urine catecholamine levels, or positivity in MIBG scan. HR-NB as defined by risk-related treatment guidelines and international criteria,102 i.e., metastatic/non-localized disease with MYCN amplification (any age), metastatic disease >18 months old, MYCN-amplified localized disease (any age), or disease resistant to standard chemotherapy. HR-NB (as defined above) and in 1) first CR at ≥ 6 months from initiation of immunotherapy using anti-GD2 antibody, or 2) second or subsequent CR (achieved after treatment for PD). CR is defined according to the International Neuroblastoma Response Criteria. Patients with positive MIBG scan but negative FDG-PET scan, and CR in 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 to hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam. Plus: Absolute neutrophil count (ANC) ≥ 500/mcl Absolute lymphocyte count ≥ 500/mcl >21 and <180 days between completion of systemic therapy and 1st vaccination. A negative pregnancy test is required for patients with child-bearing capability Signed informed consent indicating awareness of the investigational nature of this program. Exclusion Criteria: Patients with grade 4 hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam, using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA. History of allergy to KLH, QS-21, OPT-821, or glucan Prior treatment with this vaccine. Active life-threatening infection requiring systemic therapy. Inability to comply with protocol requirements.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Group 1<br>Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #5 ( 20 weeks), then only one 14-day cycle with each of vaccinations #6-#10. | Biological: OPT-821 (QS-21)<br>* Comprised of the immunological adjuvant OPT-821 (QS-21)<br>Dietary Supplement: oral β-glucan<br>* Participants will be randomized to receive this agent in two different schedules<br>|
| Experimental: Group 2<br>Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #7 ( 52 weeks), then only one 14-day cycle with each of vaccinations #8-#10. | Biological: OPT-821 (QS-21)<br>* Comprised of the immunological adjuvant OPT-821 (QS-21)<br>Dietary Supplement: oral β-glucan<br>* Participants will be randomized to receive this agent in two different schedules<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean antibody titer in ng/ml of anti-GD2 IgG1 titer | To determine the effect of oral β-glucan schedule on anti-GD2 antibody titers among patients who are in first or second (or later) CR, i.e., have no evidence of neuroblastoma by standard studies. | up to 32 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma, HR-NB, Memorial Sloan Kettering Cancer Center, 23-198