Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow

NCT03363373 · tracked via the Priya Life Science USA tracker

Sponsor

Y-mAbs Therapeutics

Phase

Phase 2

Started

2018-04-03

Last updated

2026-07-30

Condition(s) studied

Neuroblastoma

Investigational drug(s) / intervention(s)

GM-CSF + Naxitamab

GM-CSF + Naxitamab: Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Humanized IgG1 monoclonal GD2 antibody

Study summary

Children and adults diagnosed with high-risk neuroblastoma patients with primary refractory disease or incomplete response to salvage treatment in bone and/or bone marrow will be treated for up to 101 weeks with naxitamab and granulocyte-macrophage colony stimulating factor (GM-CSF). Patients will be followed for up to five years after first dose.

Naxitamab, also known as hu3F8 is a humanised monoclonal antibody targeting GD2

Eligibility

Sex

ALL

Min age

1 Year

Max age

—

Healthy volunteers

Inclusion Criteria: * Diagnosis of neuroblastoma as defined per International Neuroblastoma Response Criteria * High-risk neuroblastoma patients with either primary refractory disease or incomplete response to salvage treatment (in both cases including stable disease, minor response and partial response) evaluable in bone and/or bone marrow. * Life expectancy ≥ 6 months Exclusion Criteria: * Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks before 1st dose of GM-CSF * Evaluable neuroblastoma outside bone and bone marrow * Existing major organ dysfunction \> Grade 2, with the exception of hearing loss, hematological status, kidney and liver function * Active life-threatening infection

Primary outcome measure(s)

Response rate during Naxitamab treatment — 101 weeks
Overall objective response rate (ORR) during the Naxitamab treatment period that will be centrally assessed according to the International Neuroblastoma Response Criteria (INRC) modified with 123I-MIBG criteria and following the use of 18F FDG-PET for MIBG non-avid lesions.

Trial sites (20)

Facility	City	Region
Riley Hospital for Children	Indianapolis	Indiana
Memorial Sloan Kettering Cancer Center	New York	New York
The Hospital for Sick Children	Toronto	Canada
Rigshospitalet	Copenhagen	Denmark
University Medical Center Hamburg-Eppendorf	Hamburg	Germany
Johannes Gutenberg-Universität	Mainz	Germany
University Hospital Regensburg	Regensburg	Germany
Hong Kong Children's Hospital	Hong Kong	Hong Kong
Queen Mary Hospital	Hong Kong	Hong Kong
Tata Memorial Centre	Mumbai	India
Giannina Gaslini Hospital	Genoa	Genoa
Fondazione IRCCS Istituto Nazionale dei Tumori	Milan	Milan
Ospedale Pediatrico Bambino Gesù	Rome	Italy
Hospital Sant Joan de Déu	Barcelona	Spain
Hospital Infantil Universitario Niño Jesús	Madrid	Spain
Hospital Universitario Virgen Del Rocío	Seville	Spain
Hospital Universitario y Politécnico La Fe	Valencia	Spain
The Royal Glasgow Children's Hospital	Glasgow	United Kingdom
Leeds General Infirmary	Leeds	United Kingdom
University Hospital Southampton	Southampton	United Kingdom

Other trials for the same condition

Flavored, Oral Irinotecan VAL-413 (Orotecan®) Given With Temozolomide for Treatment of Recurrent Pediatric Solid Tumors Active, not recruiting

A Study of LY3295668 Erbumine in Participants With Relapsed/Refractory Neuroblastoma Active, not recruiting

Open-Label Study of 18F-mFBG for Imaging Neuroblastoma Active, not recruiting

Substudy 01A: Zilovertamab Vedotin in Pediatric and Young Adult Participants With Hematologic Malignancies or Solid Tumors (MK-9999-01A/LIGHTBEAM-U01) Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03363373 on ClinicalTrials.gov ↗ ← All trials in the USA