3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Primary Refractory Neuroblastoma in Bone Marrow

Memorial Sloan Kettering Cancer Center (MSK)

Status and phase: Completed; Phase 2

Conditions: Neuroblastoma

Treatments: Biological: 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic

Study type: Interventional

Funder types: Other

Identifiers

NCT01183897

09-161

Details and patient eligibility

About

The purpose of this study is to see find out what effects, good and/or bad, the combination of 3F8 and GM-CSF has on the patient and the cancer.

Enrollment: 31 patients

Sex: All

Ages: 18+ months old

Volunteers: No Healthy Volunteers

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 or MYCN-amplified stage 4S.
• Patients have primary refractory disease limited to BM, i.e., high-risk NB (defined above) resistant to standard therapy, as evidenced by incomplete response in BM, but no measurable MIBG-avid soft tissue tumor assessable for response and no progressive disease.
• 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 v 4.0) 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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31 participants in 1 patient group

3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic

Experimental group

Description:

This phase II study of the anti-GD2 murine IgG3 monoclonal antibody 3F8 combined with granulocyte-macrophage colony stimulating factor (GM-CSF) will assess response in of primary refractory neuroblastoma in bone marrow (i.e., incomplete response to standard treatment).

Treatment:

Biological: 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic