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

Memorial Sloan Kettering Cancer Center (MSK)

Status and phase: Completed; Phase 2

Conditions: Neuroblastoma

Treatments: Drug: 3F8 monoclonal antibody and 13-cis-Retinoic Acid

Study type: Interventional

Funder types: Other

Identifiers

NCT01183416

09-158

Details and patient eligibility

About

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.

Enrollment: 4 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, 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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4 participants in 1 patient group

3F8 monoclonal antibody and 13-cis-Retinoic Acid

Experimental group

Description:

This phase II, open-label, single arm trial assesses the anti-NB activity of high-dose 3F8 (80 mg/m2/day), which is used in cycles 1-2, with return to standard 3F8 dosage (20 mg/m2/day) in subsequent cycles. The patients are post-transplant and in 1st complete/very good partial remission (CR/VGPR),89 with no evidence of NB by standard studies, but are at high risk for relapse.

Treatment:

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