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A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma

Primary Purpose

Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
OPT-821 (QS-21)
oral β-glucan
Sponsored by
Memorial Sloan Kettering Cancer Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neuroblastoma focused on measuring Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma, HR-NB, Memorial Sloan Kettering Cancer Center, 23-198

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Diagnosis of NB as defined by international criteria,102 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,102 i.e., metastatic/non-localized disease with MYCN amplification (any age), metastatic disease >18 months old, MYCN-amplified 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. Plus: Absolute neutrophil count (ANC) ≥ 500/mcl Absolute lymphocyte count ≥ 500/mcl >21 and <180 days between completion of systemic therapy and 1st vaccination. A negative pregnancy test is required for patients with child-bearing capability Signed informed consent indicating awareness of the investigational nature of this program. Exclusion Criteria: Patients with 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. History of allergy to KLH, QS-21, OPT-821, or glucan Prior treatment with this vaccine. Active life-threatening infection requiring systemic therapy. Inability to comply with protocol requirements.

Sites / Locations

  • Memorial Sloan Kettering at Basking Ridge (Consent only)Recruiting
  • Memorial Sloan Kettering Monmouth (Consent Only)Recruiting
  • Memorial Sloan Kettering Bergen (Consent Only)Recruiting
  • Memorial Sloan Kettering Suffolk-Commack (Consent only)Recruiting
  • Memorial Sloan Kettering Westchester (Consent only)Recruiting
  • Memorial Sloan Kettering Cancer Center (All Protocol Activities)Recruiting
  • Memorial Sloan Kettering Nassau (Consent Only)Recruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Group 1

Group 2

Arm Description

Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #5 (~20 weeks), then only one 14-day cycle with each of vaccinations #6-#10.

Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #7 (~52 weeks), then only one 14-day cycle with each of vaccinations #8-#10.

Outcomes

Primary Outcome Measures

Mean antibody titer in ng/ml of anti-GD2 IgG1 titer
To determine the effect of oral β-glucan schedule on anti-GD2 antibody titers among patients who are in first or second (or later) CR, i.e., have no evidence of neuroblastoma by standard studies.

Secondary Outcome Measures

Full Information

First Posted
September 21, 2023
Last Updated
September 21, 2023
Sponsor
Memorial Sloan Kettering Cancer Center
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1. Study Identification

Unique Protocol Identification Number
NCT06057948
Brief Title
A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma
Official Title
Phase II Trial of a Bivalent Vaccine With the Immunological Adjuvant OPT-821 (QS-21), in Combination With Randomization of Oral β-glucan, for High-Risk Neuroblastoma
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 21, 2023 (Actual)
Primary Completion Date
March 21, 2026 (Anticipated)
Study Completion Date
March 21, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Memorial Sloan Kettering Cancer Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
The purpose of this study is to test which treatment schedule of β-glucan with bivalent vaccine is more effective for participants with high-risk neuroblastoma that is in complete remission.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma
Keywords
Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma, HR-NB, Memorial Sloan Kettering Cancer Center, 23-198

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
94 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Group 1
Arm Type
Experimental
Arm Description
Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #5 (~20 weeks), then only one 14-day cycle with each of vaccinations #6-#10.
Arm Title
Group 2
Arm Type
Experimental
Arm Description
Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #7 (~52 weeks), then only one 14-day cycle with each of vaccinations #8-#10.
Intervention Type
Biological
Intervention Name(s)
OPT-821 (QS-21)
Intervention Description
Comprised of the immunological adjuvant OPT-821 (QS-21)
Intervention Type
Dietary Supplement
Intervention Name(s)
oral β-glucan
Intervention Description
Participants will be randomized to receive this agent in two different schedules
Primary Outcome Measure Information:
Title
Mean antibody titer in ng/ml of anti-GD2 IgG1 titer
Description
To determine the effect of oral β-glucan schedule on anti-GD2 antibody titers among patients who are in first or second (or later) CR, i.e., have no evidence of neuroblastoma by standard studies.
Time Frame
up to 32 weeks

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diagnosis of NB as defined by international criteria,102 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,102 i.e., metastatic/non-localized disease with MYCN amplification (any age), metastatic disease >18 months old, MYCN-amplified 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. Plus: Absolute neutrophil count (ANC) ≥ 500/mcl Absolute lymphocyte count ≥ 500/mcl >21 and <180 days between completion of systemic therapy and 1st vaccination. A negative pregnancy test is required for patients with child-bearing capability Signed informed consent indicating awareness of the investigational nature of this program. Exclusion Criteria: Patients with 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. History of allergy to KLH, QS-21, OPT-821, or glucan Prior treatment with this vaccine. Active life-threatening infection requiring systemic therapy. Inability to comply with protocol requirements.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Brian Kushner, MD
Phone
1-833-MSK-KIDS
Email
kushnerb@MSKCC.ORG
First Name & Middle Initial & Last Name or Official Title & Degree
Fiorella Iglasias Cardenas, MD, MS
Phone
1-833-MSK-KIDS
Email
iglesiaf@mskcc.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Brian Kushner, MD
Organizational Affiliation
Memorial Sloan Kettering Cancer Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Memorial Sloan Kettering at Basking Ridge (Consent only)
City
Basking Ridge
State/Province
New Jersey
ZIP/Postal Code
07920
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Kushner, MD
Phone
833-675-5491
Facility Name
Memorial Sloan Kettering Monmouth (Consent Only)
City
Middletown
State/Province
New Jersey
ZIP/Postal Code
07748
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Kushner, MD
Phone
833-675-5491
Facility Name
Memorial Sloan Kettering Bergen (Consent Only)
City
Montvale
State/Province
New Jersey
ZIP/Postal Code
07645
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Kushner, MD
Phone
833-675-5491
Facility Name
Memorial Sloan Kettering Suffolk-Commack (Consent only)
City
Commack
State/Province
New York
ZIP/Postal Code
11725
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Kushner, MD
Phone
833-675-5491
Facility Name
Memorial Sloan Kettering Westchester (Consent only)
City
Harrison
State/Province
New York
ZIP/Postal Code
10604
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Kushner, MD
Phone
833-675-5491
Facility Name
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Kushner, MD
Phone
833-675-5491
Facility Name
Memorial Sloan Kettering Nassau (Consent Only)
City
Uniondale
State/Province
New York
ZIP/Postal Code
11553
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Kushner, MD
Phone
833-675-5491

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
Links:
URL
http://www.mskcc.org
Description
Memorial Sloan Kettering Cancer Center

Learn more about this trial

A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma

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