3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Non-Myeloablative Therapy in Patients With High-Risk Neuroblastoma

Condition(s) targeted: Neuroblastoma

Intervention: 3F8 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 find out what effects, good and/or bad, the combination of 3F8 and GM-CSF has on the patient and the cancer. Antibodies are made by the body to attack tumors and to fight infections. 3F8 is the name of one kind of antibody. It is made by mice, and it can attack neuroblastoma in people. 3F8 has been used safely in many patients, and it has killed cancer cells in some patients. One way it can kill cancer cells is by causing the patient's own white blood cells to attack the cancer. Granulocytes are one kind of white blood cell. GM-CSF increases the number of granulocytes in people, and it makes the granulocytes better able to kill the cancer cells.

Official title: 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Non-Myeloablative Therapy 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

Secondary outcome:

Apply real-time quantitative RT-PCR

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 (≥18 months of age) MYCN amplification, MYCN-amplified stage 2 or stage 3 (any age), or MYCN-amplified stage 4S.

- The patients are in first CR/VGPR after conventional therapy. They have 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: March 16, 2015