NCT04936529

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

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 286 (estimated)
• Sponsor: Memorial Sloan Kettering Cancer Center · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Accepted

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

Interventions

Type	Name	Description
DIETARY_SUPPLEMENT	β-glucan	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.
DRUG	GM-CSF	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.
BIOLOGICAL	OPT-821	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.

Timeline

• Start date: 2021-08-02
• Primary completion: 2026-06-15
• Completion: 2026-06-15
• First posted: 2021-06-23
• Last updated: 2026-03-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study