A Study of the Effect of Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin in Patients in First Remission of High-Risk Neuroblastoma

Memorial Sloan Kettering Cancer Center (MSK)

Status and phase: Completed; Phase 2

Conditions: Neuroblastoma

Treatments: Drug: Isotretinoin; Biological: Hu3F8; Drug: GM-CSF

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03033303

16-1643

Details and patient eligibility

About

The purpose of this study is to test see the combined effects of the study drug called Humanized 3F8 (Hu3F8) when used with granulocyte-macrophage colony stimulating factor (GM-CSF). Hu3F8 plus GM-CSF could prevent your neuroblastoma from growing, but it could also cause side effects.

Enrollment: 59 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of NB as defined by a) histopathology (confirmed by the MSK Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels.
• Patients must have high-risk NB (MYCN-amplified stage 2/3/4/4S of any age and MYCN-nonamplified stage 4 in patients greater than 18 months of age).
• Patients must be in first CR/VGPR
• Patients must have a negative human anti-hu3F8 antibody (HAHA) titer

Exclusion criteria

• Existing major organ dysfunction, i.e., renal., cardiac, hepatic, neurologic, pulmonary, or gastrointestinal toxicity ≥ grade 3, with the exception of hearing loss and hematologic toxicity.
• Active life-threatening infection.
• 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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59 participants in 1 patient group

Hu3F8/GM-CSF Plus Isotretinoin

Experimental group

Description:

In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF x5 cycles and isotretinoin x6 cycles. Isotretinoin starts after cycle 2 of hu3F8/GM-CSF.

Treatment:

Drug: GM-CSF

Biological: Hu3F8

Drug: Isotretinoin