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

NCT03033303 | Completed Phase 2 FDA Drug

• 1 other identifier: 16-1643
• Study Type: interventional
• Target: 59
• Locations: 1 country
• Sites: 1

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.

Conditions

Neuroblastoma

Keywords

Hu3F8; GM-CSF; Isotretinoin; 16-1643

Outcome Measures

Primary Outcomes (1)

• relapse-free survival (RFS)

  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.

  2 years

Study Arms (1)

Hu3F8/GM-CSF Plus Isotretinoin

EXPERIMENTAL

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.

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

Interventions

Hu3F8 BIOLOGICAL

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.

Hu3F8/GM-CSF Plus Isotretinoin

GM-CSF DRUG

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

Hu3F8/GM-CSF Plus Isotretinoin

Isotretinoin DRUG

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

Hu3F8/GM-CSF Plus Isotretinoin

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• Y-mAbs Therapeutics: collaborator

Study Sites (1)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Related Publications (1)

• Kushner BH, Modak S, Mauguen A, Basu EM, Kramer K, Roberts SS, Cheung IY, Cheung NV. A Phase II Trial of Naxitamab plus Stepped-up Dosing of GM-CSF for Patients with High-Risk Neuroblastoma in First Complete Remission. Clin Cancer Res. 2025 May 15;31(10):1877-1884. doi: 10.1158/1078-0432.CCR-24-3427.

  PMID: 40067131 DERIVED

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Conditions

Neuroblastoma

Interventions

naxitamab; Granulocyte-Macrophage Colony-Stimulating Factor; Isotretinoin

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Retinoids; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Pigments, Biological

Study Officials

• Brian H. Kushner, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

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

Study Record Dates

• First Submitted: January 24, 2017
• First Posted: January 26, 2017
• Study Start: January 23, 2017
• Primary Completion: September 29, 2025
• Study Completion: September 29, 2025
• Last Updated: October 7, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)