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

Launched by MEMORIAL SLOAN KETTERING CANCER CENTER · Jun 16, 2021

Keywords

ClinConnect Summary

This clinical trial is testing a new treatment approach for children and young adults with high-risk neuroblastoma, a type of cancer. The study is looking at a combination of a special vaccine, a sugar called beta-glucan, and a protein called GM-CSF. The goal is to see if this combination can help strengthen the immune system to fight any remaining cancer cells after the patients have completed their initial treatment and are in complete remission.

To be eligible for this trial, participants need to have been diagnosed with high-risk neuroblastoma and must be in a stable condition after prior treatments. Key requirements include having certain blood counts within a safe range and having completed other cancer therapies at least three weeks before starting this study. Participants can expect to receive the vaccine and other components in a controlled environment, while doctors monitor their health closely. It’s important to note that anyone who joins the trial will need to agree to the study's guidelines and may have to undergo some tests to ensure they are suitable for participation.

Gender

ALL

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 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