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

Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin for Consolidation of First Remission of Patients With High-Risk Neuroblastoma: A Phase II Study

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03033303

Enrollment

Registered

2017-01-26

Start date

2017-01-23

Completion date

2025-09-29

Last updated

2025-10-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neuroblastoma

Keywords

Hu3F8, GM-CSF, Isotretinoin, 16-1643

Brief summary

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.

Interventions

BIOLOGICALHu3F8

Day 1: hu3F8 infused iv over \ 30 to 90 minutes. Day 3: hu3F8 infused iv over \ 30 to 90 minutes. Day 5: hu3F8 infused iv over \ 30 to 90 minutes.

DRUGGM-CSF

Days -4 to 0: GM-CSF 250 mcg/m2/day, subcutaneously. Days 1 to 5: GM-CSF 500 mcg/m2/day, subcutaneously.

DRUGIsotretinoin

Isotretinoin is administered at 160 mg/m2/d, divided into two doses, x14 days.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Intervention model description

In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF.

Eligibility

Sex/Gender

ALL

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

Design outcomes

Primary

Measure	Time frame	Description
relapse-free survival (RFS)	2 years	Patients are considered a response failure under this protocol if progressive disease is evident before two years. Deaths from toxicity attributable to protocol treatment will be counted as events. Patients who withdraw or are lost to followup before two years will be considered as progressions.

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026