New vaccine combo aims to outsmart aggressive childhood cancer
NCT ID NCT04936529
First seen Jun 27, 2026 · Last updated Aug 12, 2026 · Updated 4 times
Summary
This study tests whether a bivalent vaccine, taken with a sugar called beta-glucan and a protein (GM-CSF), can help prevent high-risk neuroblastoma from returning in children who are currently in remission. The treatment works by training the immune system to recognize and attack cancer cells. About 286 participants will be enrolled to see if adding GM-CSF improves the immune response.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 286 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2021
- Expected to finish
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Jun 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 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 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 allowed but 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 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 Criteria: * 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Memorial Sloan Kettering Cancer Center
RECRUITINGNew York, New York, 10065, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a Two-Drug combo outsmart returning childhood cancers?
- Can a Two-Drug combo beat tough childhood cancers?
- A simple blood test may reveal hidden infertility risk in young cancer survivors
- Can a shorter chemo combo tame High-Risk neuroblastoma?
- Can a radioactive 'Smart Bomb' take down resistant childhood cancers?