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

NCT03363373 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 201
• Study Type: interventional
• Target: 122
• Locations: 10 countries
• Sites: 26

Brief 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

Conditions

Neuroblastoma

Keywords

Antibody, Neuroblastoma, Pediatric, Adult

Outcome Measures

Primary Outcomes (1)

• Response rate during Naxitamab treatment

  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.

  101 weeks

Secondary Outcomes (20)

• Incidence of adverse events and serious adverse events

  101 weeks

• Duration of Response (DoR)

  101 weeks

• Complete Response Rate

  101 weeks

• Assessment of the maximum serum concentration (cmax) of naxitamab

  Pre-naxitamab dose - 552 hours

• Assessment of the minimum serum concentration (cmin) of naxitamab

  Pre-naxitamab dose - 552 hours

Study Arms (1)

GM-CSF + Naxitamab

EXPERIMENTAL

Each investigational cycle is started with 5 days of GM-CSF administered at 250 µg/m2/day in advance of the start of Naxitamab administration. GM-CSF is thereafter administered at 500 µg/m2/day on days 1 to 5. As standard treatment, Naxitamab is administered at 3 mg/kg/day on days 1, 3, and 5 totalling 9 mg/kg per cycle. Treatment cycles are repeated every 4 weeks until CR or PR followed by 5 additional cycles every 4 weeks (±1 week). Subsequent cycles are repeated every 8 weeks (±2 weeks) through 101 weeks from first infusion at the discretion of the investigator. After end of treatment patients will enter a long-term follow up for up to 3 years after end of treatment visit.

Biological: GM-CSF + Naxitamab

Interventions

GM-CSF + Naxitamab BIOLOGICAL

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

GM-CSF + Naxitamab

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Y-mAbs Therapeutics: lead

Study Sites (26)

University of Florida

Gainesville, Florida, 32611, United States

WITHDRAWN

University of Chicago

Chicago, Illinois, 60637, United States

WITHDRAWN

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

ACTIVE NOT RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

ACTIVE NOT RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

WITHDRAWN

M.D. Anderson Cancer Center

Houston, Texas, 77030, United States

WITHDRAWN

The Hospital for Sick Children

Toronto, M5G 1X8, Canada

RECRUITING

Rigshospitalet

Copenhagen, 2100, Denmark

RECRUITING

Hopital pour enfants de la Timone

Marseille, Marseille, 13005, France

WITHDRAWN

University Medical Center Hamburg-Eppendorf

Hamburg, Germany

RECRUITING

Johannes Gutenberg-Universität

Mainz, Germany

RECRUITING

University Hospital Regensburg

Regensburg, Germany

RECRUITING

Hong Kong Children's Hospital

Hong Kong, Hong Kong

RECRUITING

Queen Mary Hospital

Hong Kong, Hong Kong

ACTIVE NOT RECRUITING

Tata Memorial Centre

Mumbai, 400012, India

RECRUITING

Giannina Gaslini Hospital

Genoa, Genoa, 16147, Italy

RECRUITING

Fondazione IRCCS Istituto Nazionale dei Tumori

Milan, Milan, 20133, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesù

Rome, Italy

RECRUITING

Hospital Sant Joan de Déu

Barcelona, 08950, Spain

RECRUITING

Hospital Infantil Universitario Niño Jesús

Madrid, 28009, Spain

RECRUITING

Hospital Universitario Virgen Del Rocío

Seville, Spain

RECRUITING

Hospital Universitario y Politécnico La Fe

Valencia, 46026, Spain

RECRUITING

The Royal Glasgow Children's Hospital

Glasgow, G51 4TF, United Kingdom

RECRUITING

Leeds General Infirmary

Leeds, LS1 3EX, United Kingdom

RECRUITING

The Royal Marsden

London, SW3 6JJ, United Kingdom

WITHDRAWN

University Hospital Southampton

Southampton, SO16 6YD, United Kingdom

RECRUITING

MeSH Terms

Conditions

Neuroblastoma

Interventions

Granulocyte-Macrophage Colony-Stimulating Factor; naxitamab

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

Central Study Contacts

Rhiannon Wright

CONTACT

+447969051167; clinicaltrials@ymabs.com

Study Design

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

Model Details: Patients will receive cycles of GM-CSF and Naxitamab every 4 weeks up to a total of 101 weeks. Safety and efficacy will be investigated with short-term follow-up at minimum 4 weeks after last treatment and with long-term follow-up for up to 3 years after end of treatment visit.

Study Record Dates

• First Submitted: November 6, 2017
• First Posted: December 6, 2017
• Study Start: April 3, 2018
• Primary Completion: April 1, 2026
• Study Completion (Estimated): April 1, 2028
• Last Updated: February 19, 2026

Record last verified: 2026-02

Locations

IT (3); CA (1); DE (3); IN (1); US (6); HK (2); ES (4); DK (1); FR (1); GB (4)