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

NCT04936529 | Recruiting Phase 2 FDA Drug

• 1 other identifier: 21-206
• Study Type: interventional
• Target: 286
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to explore the combination of a bivalent vaccine, a sugar called beta-glucan (β-glucan), and a protein called granulocyte-macrophage colony stimulating factor (GM-CSF) as an effective treatment for people with high-risk neuroblastoma that is in complete remission. The combination may be effective because the different parts of the treatment work to strengthen the immune system's response against cancer cells in different ways.

Conditions

Neuroblastoma

Keywords

Neuroblastoma; High-risk Neuroblastoma; OPT-821; QS-21; β-glucan; GM-CSF; Memorial Sloan Kettering Cancer Center; 21-206

Outcome Measures

Primary Outcomes (1)

• Effect of GM-CSF on anti-GD2 antibody titers

  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.

  32 weeks

Study Arms (3)

Group 1

EXPERIMENTAL

Group 1 participants receive oral β-glucan (40 mg/kg/day x 14 days) starting week 1. This schedule includes annual booster vaccinations, with β-glucan, administered at weeks 8, 20, 32, 52, 78, 104, and 156 (vaccinations #1 \& #4-10). Participants will not receive GM-CSF.

Dietary Supplement: β-glucan; Biological: OPT-821

Group 2

EXPERIMENTAL

Group 2 participants receive oral β-glucan (40 mg/kg/day) starting week 1. Participants also receive GM-CSF (250 mcg/m2/day) x3 days with vaccinations #1-#3; x7 days with vaccinations #4-#9; and x5 days with vaccination #10. The treatment includes annual booster vaccinations, with a 2-week cycle of β-glucan, administered at weeks 8, 20, 32, 52, 78, 104, and 156 (vaccinations #1 \& #4-10)

Dietary Supplement: β-glucan; Drug: GM-CSF; Biological: OPT-821

Group 3

EXPERIMENTAL

Group 3 will include participants who cannot be randomized (e.g., due to allergy to GMCSF). It will also include participants previously treated with this vaccine and oral β-glucan on the predecessor MSK protocol IRB# 05-075 or on this protocol (participants can therefore be enrolled more than one time on this protocol). These participants will be treated as in Group 1. Participants who are registered to Group 3 and have been previously treated with vaccine (in this protocol or MSK predecessor 05-075) will not receive vaccines 4 and 6. These patients will receive a total of 8 injections. The analyses in this group will be exploratory.

Dietary Supplement: β-glucan; Biological: OPT-821

Interventions

OPT-821 BIOLOGICAL

Vaccine injections must occur a minimum of 6 days apart. After the first four vaccine injections, vaccines can be administered up to two weeks earlier or later than indicated without representing a protocol violation.

Also known as: QS-21

Group 1; Group 2; Group 3

β-glucan DIETARY_SUPPLEMENT

Group 1 participants receive oral β-glucan (40 mg/kg/day) starting week 1 and continue with \~2 weeks on, \~2 weeks off, up to 1 cycle after vaccination #7, then 1 cycle with each subsequent vaccination (#8-#14). This schedule includes annual booster vaccinations, with a 2-week cycle of β-glucan, administered at months 36 (3 years), 48 (4 years), and 60 (5 years). Group 2 participants receive oral β-glucan (40 mg/kg/day) starting week 1 and continue with \~2 weeks on, \~2 weeks off, up to 1 cycle after vaccination #7, then 1 cycle with each subsequent vaccination (#8-#14). Group 3 participants will be treated as in Group 1.

Also known as: oral β-glucan

Group 1; Group 2; Group 3

GM-CSF DRUG

Group 1 participants will not receive GM-CSF. Group 2 participants will receive GM-CSF (250 mcg/m2/day) x3 days with vaccinations #1-#3; x7 days with vaccinations #4-#11; and x5 days with vaccinations #12-#14. Group 3 participants will be treated as in Group 1.

Group 2

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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
• \- Hepatic Function
• Serum bilirubin ≤ 3.0 × ULN
• Aspartate transaminase (AST) ≤ 5.0 × ULN
• Alanine aminotransferase (ALT) ≤ 5.0 × ULN

You may not qualify if:

• 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

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead

Study Sites (1)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

RECRUITING

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Conditions

Neuroblastoma

Interventions

Granulocyte-Macrophage Colony-Stimulating Factor; saponin QA-21V1

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Brian Kushner, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Brian Kushner, MD

CONTACT

1-833-675-5437; kushnerb@mskcc.org

Fiorella Iglesias Cardenas, MD, MS

CONTACT

1-833-675-5437

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 16, 2021
• First Posted: June 23, 2021
• Study Start: August 2, 2021
• Primary Completion: June 15, 2026
• Study Completion: June 15, 2026
• Last Updated: May 7, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

US (1)