Phase I/II Trial of a Bivalent Vaccine With Escalating Doses of the Immunological Adjuvant OPT-821, in Combination With Oral ß-glucan for High-Risk Neuroblastoma
- Diagnosis of neuroblastoma (NB) as defined by international criteria, i.e.,
histopathology (confirmed by the MSKCC Department of Pathology) or bone marrow
metastases plus high urine catecholamine levels.
- High-risk NB as defined by risk-related treatment guidelines and the International NB
Staging System, i.e., stage 4 with (any age) or without (>18 months old) MYCN
amplification, MYCN-amplified stage 3 (unresectable; any age), MYCN-amplified stage
4S, or disease resistant to standard chemotherapy.
- Relapsed high-risk NB (as defined above) and now in second or subsequent remission.
Remission is defined as complete (CR) or very good partial (VGPR)remission,
according to the International Neuroblastoma Response Criteria. Urine
catecholamine levels are no longer taken into consideration when staging.
Patients can be considered as in VGPR with 1 or 2 MIBG (+) sites that were previously-
- Absolute lymphocyte count > or = to 500/mcl and absolute neutrophil count > or = to
- Creatinine ≤ 2.0 mg/dL
- ALT, AST and Alkaline Phosphatase ≤ 2.5 times the upper limit of normal
- Bilirubin ≤ 2.0 mg/dL
- Patients with less than grade 3 toxicities (using the CTCAE v3.0) related to cardiac,
neurological, pulmonary or gastrointestinal function as determined by physical exam.
- Prior treatment with other immunotherapy, including antibodies, is allowed
- > or = to 3 weeks between completion of systemic therapy and 1st vaccination.
- Signed informed consent indicating awareness of the investigational nature of this
- History of allergy to KLH, QS-21, OPT-821, or glucan.
- Active life-threatening infection.
- Inability to comply with protocol requirements.