Clinical Trials /

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

NCT04936529

Description:

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.

Related Conditions:
  • Neuroblastoma
Recruiting Status:

Not yet recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: A Study of a Vaccine in Combination With β-glucan and GM-CSF in People With Neuroblastoma
  • Official Title: 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

Clinical Trial IDs

  • ORG STUDY ID: 21-206
  • NCT ID: NCT04936529

Conditions

  • Neuroblastoma

Interventions

DrugSynonymsArms
GM-CSFGroup 2
OPT-821QS-21Group 1

Purpose

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.

Trial Arms

NameTypeDescriptionInterventions
Group 1ExperimentalGroup 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). Participants will not receive GM-CSF.
  • OPT-821
Group 2ExperimentalGroup 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). Participants also receive GM-CSF (250 mcg/m2/day) x3 days with vaccinations #1-#3; x7 days with vaccinations #8-#11; and x5 days with vaccinations #12-#14. The treatment 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).
  • GM-CSF
  • OPT-821
Group 3ExperimentalGroup 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. The analyses in this group will be exploratory.
  • OPT-821

Eligibility Criteria

        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.

          -  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.

          -  Absolute lymphocyte count >500/mcl and absolute neutrophil count ≥ 500/mcl.

          -  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
             cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as
             determined by blood tests or physical exam.

          -  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 with history of allergy to GM-CSF or who are unable to obtain GM-CSF because
             of insurance issues are eligible but will be assigned to Group 3 (no GM-CSF
             exploratory arm).

          -  Patients previously enrolled on this trial are eligible for repeat enrollment but will
             be assigned to Group 3 and will not be included in the primary biostatistical
             analyses.

          -  Signed informed consent indicating awareness of the investigational nature of this
             program.

        Exclusion Criteria:

          -  History of allergy to KLH, QS-21, OPT-821, or glucan.

          -  Active life-threatening infection.

          -  Inability to comply with protocol requirements.
      
Maximum Eligible Age:N/A
Minimum Eligible Age:N/A
Eligible Gender:All
Healthy Volunteers:Accepts Healthy Volunteers

Primary Outcome Measures

Measure:Effect of GM-CSF on anti-GD2 antibody titers
Time Frame:32 weeks
Safety Issue:
Description: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.

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Not yet recruiting
Lead Sponsor:Memorial Sloan Kettering Cancer Center

Trial Keywords

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

Last Updated

July 2, 2021