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

Memorial Sloan Kettering Cancer Center (MSK)

Status and phase: Completed; Phase 2

Conditions: Neuroblastoma

Treatments: Drug: 3F8 and 13-cis-retinoic acid

Study type: Interventional

Funder types: Other

Identifiers

NCT01183429

09-159

Details and patient eligibility

About

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.

Enrollment: 39 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 (≥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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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39 participants in 1 patient group

3F8 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. Clinical results will be compared to those in the predecessor trials which used only the standard 3F8 dosage. Starting with A(8), patients no longer receive high dose 3F8 but receive only standard dose 3F8 (20mg/m2/day) for all cycles.

Treatment:

Drug: 3F8 and 13-cis-retinoic acid