Vaccine combined with β-glucan and GM-CSF for treating high-risk 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
This study is testing a new vaccine combined with two other treatments to see if it can help people with high-risk neuroblastoma who are in complete remission stay cancer-free longer.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 286 (estimated) |
| Sex | All |
| Sponsor | Memorial Sloan Kettering Cancer Center Academic / other |
| Drugs / interventions | chemotherapy, immunotherapy |
| Locations | 1 site (New York, New York) |
| Trial ID | NCT04936529 on ClinicalTrials.gov |
What this trial studies
This study investigates the effectiveness of a bivalent vaccine in combination with β-glucan and GM-CSF for patients with high-risk neuroblastoma who are in complete remission. The approach aims to enhance the immune system's ability to target and eliminate cancer cells through a multi-faceted treatment strategy. Participants will receive the combination therapy to assess its safety and efficacy in improving outcomes for this challenging condition.
Who should consider this trial
Good fit: Ideal candidates are individuals diagnosed with high-risk neuroblastoma who are in complete remission after prior treatments.
Not a fit: Patients with low-risk neuroblastoma or those who are not in complete remission may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve survival rates and quality of life for patients with high-risk neuroblastoma.
How similar studies have performed: Other studies have shown promise in using immunotherapy approaches for neuroblastoma, suggesting potential for success with this combination treatment.
Eligibility criteria
Show full inclusion / exclusion 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 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 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
Where this trial is running
New York, New York
- Memorial Sloan Kettering Cancer Center — New York, New York, United States (Recruiting)
Study contacts
- Principal investigator: Brian Kushner, MD — Memorial Sloan Kettering Cancer Center
- Study coordinator: Brian Kushner, MD
- Email: kushnerb@mskcc.org
- Phone: 1-833-675-5437
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.