High-Dose 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Myeloablative Therapy and Autologous Stem-Cell Transplantation

Condition(s) targeted: Neuroblastoma

Intervention: 3F8 monoclonal antibody and 13-cis-Retinoic Acid (Drug)

Phase: Phase 2

Status: Active, not recruiting

Sponsored by: Memorial Sloan Kettering Cancer Center

Official(s) and/or principal investigator(s):
Brian Kushner, MD, Principal Investigator, Affiliation: Memorial Sloan Kettering Cancer Center

The purpose of this study is to see if high-dose 3F8 combined with GM-CSF is better than standard dose 3F8 in treating neuroblastoma. Another purpose of the study is to find out what effects, good and/or bad, 3F8 has on cancer. The investigators also want to see if the antibody works against a very small amount of neuroblastoma (minimal residual disease) that is left in the bone marrow.

Official title: High-Dose 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Myeloablative Therapy and Autologous Stem-Cell Transplantation in Patients With High-Risk Neuroblastoma: A Phase II Study

Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Assess the impact of high-dose 3F8/GM-CSF on relapse-free survival

Secondary outcome:

Apply real-time quantitative RT-PCR to test the hypothesis that the minimal residual disease content of bone marrow

Monitor safety of the high-dose antibody treatment

Minimum age: 18 Months. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Diagnosis of NB as defined by a) histopathology (confirmed by the MSKCC Department of

Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels.

- High-risk NB as defined by risk-related treatment guidelines1 and the International

NB Staging System,89 i. e., stage 4 with (any age) or without (> or = to 18 months of age) MYCN amplification, MYCN-amplified stage 2 or stage 3 (any age), or MYCN-amplified stage 4S.

- The patients are post-stem cell transplantation and in first CR/VGPR, including no

measurable MIBG-avid soft tissue tumor assessable for response.

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

program. Exclusion Criteria:

- Creatinine > 3. 0 mg/dL

- ALT, AST and Alkaline Phosphatase > 5. 0 times the upper limit of normal

- Bilirubin > 3. 0 mg/dL

- Patients with grade 3 or higher toxicities (using the CTCAE v3. 0) related to cardiac,

neurological, pulmonary or gastrointestinal function as determined by physical exam. Patients must have normal blood pressure for age.

- Progressive disease

- History of allergy to mouse proteins.

- Active life-threatening infection.

- Human anti-mouse antibody (HAMA) titer >1000 Elisa units/ml.

- Inability to comply with protocol requirements

Memorial Sloan-Kettering Cancer Center, New York, New York 10065, United States

Memorial Sloan-Kettering Cancer Center

Starting date: August 2010
Last updated: December 16, 2014