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A Study of a Vaccine, β-glucan, and GM-CSF for Neuroblastoma

This study is looking at a combination treatment for people with high-risk neuroblastoma (a type of cancer that starts in nerve cells) who are in complete remission. The treatment includes a vaccine called OPT-821, a sugar called β-glucan, and a protein called GM-CSF. Researchers believe this combination may strengthen your immune system to fight cancer cells. The study will enroll 286 participants and aims to see how GM-CSF affects the body's immune response (measured by anti-GD2 antibody titers) at 32 weeks. You may be able to join if you have a diagnosis of high-risk neuroblastoma as defined by international criteria.

Study design
This interventional study plans to enroll 286 participants. Participants will be divided into different groups to receive varying combinations of β-glucan, GM-CSF, and the OPT-821 vaccine.
What's involved
Participants in Group 1 will take oral β-glucan in cycles, continuing up to one cycle after vaccination #7, then with each subsequent vaccination. Participants in Group 2 will receive GM-CSF injections for a set number of days with vaccinations #1-#14. All participants will receive OPT-821 vaccine injections, at least 6 days apart.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint, effect of GM-CSF on anti-GD2 antibody titers, will be measured at 32 weeks.

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NCT04936529

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

Recruiting
PHASE2All AgesInterventionalTreatment
Memorial Sloan Kettering Cancer Center
~286 participants
Updated 2026-08-11 on ClinicalTrials.gov
What's tested:β-glucanGM-CSFOPT-821

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Effect of GM-CSF on anti-GD2 antibody titers
Measured over 32 weeks

Conditions

Neuroblastoma

Explore related trials

NeuroblastomaMYCN Gene

Where it's being run

1 sites across 1 states
New York1

Study leadership

  • Brian Kushner, MD · PRINCIPAL_INVESTIGATOR · Memorial Sloan Kettering Cancer Center

Who to contact

Brian Kushner, MD
[email protected]
1-833-675-5437
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Diagnosis of NB as defined by international criteria, 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,i.e., metastatic/non-localized disease with MYCN amplification (any age), MYCN-non-amplified metastatic disease \>18 months old, MYCNamplified 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. For those patients with a positive MIBG scan, the negative FDG-PET scan can be per PI discretion to repeat within the 45 day window period prior to enrollment.
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.
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
eGFR \>60 mL/min/1.73 m\^2
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 allowed but must be completed ≥ 21 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 prior therapies.
Patients previously enrolled on this trial are eligible for repeat enrollment if they did not complete all vaccine injections during the first time on protocol but they will be assigned to Group 3 and will not be included in the primary biostatistical analyses.
A negative pregnancy test is required for patients w ith child-bearing capability.
Signed informed consent indicating awareness of the investigational nature of this program.

Exclusion

Patients w ith significant (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 (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-CSF because of insurance issues are ineligible

What this trial measures

  • Effect of GM-CSF on anti-GD2 antibody titers32 weeks

    Effect of GM-CSF on anti-GD2 antibody titers among patients who are in first or second (or later) CR, i.e., have no evidence of NB by standard studies.