Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

January 30, 2026 updated by: Giselle Sholler

A Phase II Study of Naxitamab Added to Induction Therapy for Subjects With Newly Diagnosed High-Risk Neuroblastoma

This is a prospective, multicenter clinical trial in subjects with newly diagnosed high-risk neuroblastoma to evaluate the efficacy and safety of administering naxitamab with standard induction therapy. The initial chemotherapy will include 5 cycles of multi-agent chemotherapy. Naxitamab will be added to all 5 Induction cycles. We hypothesize that the addition of anti-GD2 therapy to induction chemotherapy will result in improved end of induction responses and improved survival.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a prospective, multicenter clinical trial in subjects with newly diagnosed high-risk neuroblastoma to evaluate the efficacy and safety of administering naxitamab with standard induction therapy.

All subjects will be followed for disease response, event free survival, overall survival and toxicity. Extent of disease will be measured and assessed for changes throughout the course of the study. All efficacy analyses will be performed on the evaluable population which will consist of all enrolled subjects (subjects who initiate treatment with naxitamab in combination with GM-CSF plus standard induction therapy) and who have measurable disease at baseline.

The initial chemotherapy Induction regimen will utilize sequential administration of 5 cycles of multi-agent chemotherapy. Naxitamab will be added to all 5 Induction cycles.

Stem cell mobilization and collection will occur after the 2nd cycle of induction.

Surgical resection of the primary tumor will ideally occur after the 4th cycle of Induction but may be delayed until after the 5th cycle of Induction if medically necessary.

Disease status evaluations will occur at the following time points: (1) pre-treatment, (2) post Cycle 2 Induction (3) Prior to surgical resection (if performed), (4) End of Induction (which includes surgery and 5 cycles of chemotherapy), and (5) End of Additional/Salvage Therapy as needed.

The current standard of care for high-risk neuroblastoma involves 5-7 cycles of induction chemotherapy with surgical removal of the tumor after 4-5 cycles of chemotherapy, followed by high-dose chemotherapy plus autologous stem cell transplant, then radiation to the primary tumor bed, followed by anti-GD2 immunotherapy and cis retinoic acid. This results in a less than 60% disease free survival for high-risk NB, a survival rate that still greatly needs improvement. Two areas in which improvements can be made include: 1) to improve response rate to induction chemotherapy and 2) to improve EFS by improving maintenance therapy to prevent relapse.

We hypothesize that the addition of anti-GD2 therapy to induction chemotherapy will result in improved end of induction responses and improved survival.

Study Type

Interventional

Enrollment (Estimated)

93

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35201
        • Recruiting
        • University of Alabama/Children's of Alabama
        • Contact:
        • Principal Investigator:
          • Elizabeth Alva
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Recruiting
        • Arkansas Children's Hospital
        • Principal Investigator:
          • Kevin Bielamowicz
        • Contact:
    • California
      • Oakland, California, United States, 94609
        • Recruiting
        • UCSF Benioff Children's Hospital Oakland
        • Contact:
        • Principal Investigator:
          • Jennifer Michlitsch
      • San Diego, California, United States, 92123
        • Recruiting
        • Rady Children's Hospital
        • Contact:
        • Principal Investigator:
          • William Roberts
    • Connecticut
      • Hartford, Connecticut, United States, 06106
    • Florida
      • Gainesville, Florida, United States, 32611
        • Recruiting
        • University of Florida
        • Contact:
        • Principal Investigator:
          • Joanne Lagmay
      • Miami, Florida, United States, 33155
      • Orlando, Florida, United States, 32806
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Recruiting
        • Augusta University Health
        • Contact:
        • Principal Investigator:
          • Coleen McDonough
    • Hawaii
      • Honolulu, Hawaii, United States, 96813
        • Recruiting
        • Kapiolani Medical Center for Women and Children
        • Principal Investigator:
          • Kelley Hutchins
        • Contact:
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Recruiting
        • Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine
        • Principal Investigator:
          • Michael Ferguson
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Recruiting
        • Children's Hospital and Clinics of Minnesota
        • Contact:
        • Principal Investigator:
          • Jawhar Rawwas
    • Missouri
      • St Louis, Missouri, United States, 63104
        • Recruiting
        • Cardinal Glennon Children's Hospital
        • Principal Investigator:
          • William Ferguson
        • Contact:
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
      • Winston-Salem, North Carolina, United States, 27157
        • Withdrawn
        • Wake Forest University Health Sciences
    • Oregon
      • Portland, Oregon, United States, 97227
        • Recruiting
        • Randall Children's Hospital
        • Contact:
        • Principal Investigator:
          • Jason Glover
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina
        • Contact:
        • Principal Investigator:
          • Jaqueline Kraveka
    • Texas
      • Austin, Texas, United States, 78723
        • Recruiting
        • Dell Children's Blood and Cancer Center
        • Contact:
        • Principal Investigator:
          • Virginia Harrod
    • Virginia
      • Richmond, Virginia, United States, 23284
        • Recruiting
        • Virginia Commonwealth University
        • Contact:
        • Principal Investigator:
          • Madhu Gowda

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 17 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Diagnosis: Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. Subjects with the following disease stages at diagnosis are eligible, if they meet the other specified criteria:

  2. Subjects with newly diagnosed neuroblastoma with INRGSS Stage M disease with either of the following features:

    1. MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
    2. 365 days to ≥ 547 days of age without MYCN amplification, but unfavorable biologic features such as unfavorable histology (INPC) or diploid tumor (DNA index=1) or the presence of any segmental chromosome aberration (SCA) (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q); OR
    3. Age > 547 days of age regardless of biologic features

    Subjects with newly diagnosed neuroblastoma with INRGSS Stage MS disease with either of the following:

    1. MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals); OR
    2. 365 days to ≥ 547 days (18 months) of age without MYCN amplification, but unfavorable biologic features such as unfavorable histology (INPC) or diploid tumor (DNA index=1) or SCA as above

    Subjects with newly diagnosed neuroblastoma INRGSS Stage L2 disease with either of the following:

    1. MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals); OR
    2. 18 months to <5 years of age without MYCN amplification, but with unfavorable histology (INPC); OR
    3. ≥5 years of age without MYCN amplification, but with undifferentiated or poorly differentiated INPC Subjects with newly diagnosed neuroblastoma INRGSS Stage L1 disease that is incompletely resected with MYCN amplification.

    Subjects > 547 days of age initially diagnosed with INRGSS Stage L1, L2 or MS disease who progressed to Stage M without prior chemotherapy may enroll within 4 weeks of progression to Stage M.

    Subjects ≥ 365 days of age initially diagnosed with MYCN amplified INRGSS Stage L1 disease who progress to Stage M without systemic therapy may enroll within 4 weeks of progression to Stage M.

  3. Subjects must be age ≤ 21 years at initial diagnosis.
  4. Subjects must be >12 months of age at enrollment.

  5. Adequate cardiac function defined as:

    1. Shortening fraction of ≥ 27% by echocardiogram, or
    2. Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.

  6. Adequate liver function must be demonstrated, defined as:

    1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
    2. ALT (SGPT) < 5 x upper limit of normal (ULN) for age

  7. Subjects must have adequate renal function defined as an estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70.

    The Bedside Schwartz equation is: [(0.413) X (Height in cm)] / SCr

  8. A negative serum pregnancy test is required for female participants of childbearing potential (≥13 years of age or after onset of menses)
  9. Both male and female post-pubertal study subjects must be willing to use a highly effective contraceptive method (i.e., achieves a failure rate of <1% per year when used consistently and correctly) from the time of informed consent until 6 months after study treatment discontinuation. Such methods include: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner, sexual abstinence.
  10. Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines.

Exclusion Criteria:

  1. Subjects who are less than 1 year of age
  2. Subjects who are 12-18 months of age with INRGSS Stage M and all stage L2 subjects with favorable biologic features (i.e., nonamplified MYCN, favorable pathology, and DNA index > 1) are not eligible.
  3. Subjects who have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy.
  4. Treatment with immunosuppressive treatment (topical, inhaled and short-term emergency steroids excluded) within 4 weeks prior to enrollment
  5. Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and/or chronic oxygen requirement. In addition, room air pulse oximetry < 94% and/or abnormal pulmonary function tests if these assessments are clinically indicated.
  6. Pregnant or breastfeeding (NOTE: breast milk cannot be stored for future use while the mother is being treated on study.)
  7. Subjects receiving any investigational drug concurrently.
  8. Subjects with any other medical condition, including but not limited to malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study
  9. Subjects with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of investigational medicinal products (IMPs) or to significantly increase the severity of the toxicities experienced from trial treatment.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: HRNB Newly diagnosed subjects

5 cycles of standard of care induction + naxitimab

Naxitimab on Days 1, 3, and 5 of each cycle
Drug: Naxitamab
Naxitamab is a humanized (IgG1) anti-GD2 (hu3F8) monoclonal antibody for the treatment of neuroblastoma, osteosarcoma and other GD2-positive cancers. Naxitamab was granted accelerated approval by the FDA in 2020 as treatment (in combination with granulocyte-macrophage colony-stimulating factor - GM-CSF) for pediatric patients at least one year of age and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow demonstrating a partial response, minor response, or stable disease to prior therapy
Other Names:
  • Danyelza

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with Complete Response (CR) rate per 1993 INRC guidelines
Time Frame: 6 to 12 months
Measured by the presence of radiologically assessable disease by cross-sectional computed tomography (CT) or Magnetic resonance imaging (MRI) imaging and/or by metaiodobenzylguanidine (MIBG) or positron emission tomography (PET) scans and bone marrow (BM) response for subjects with newly-diagnosed high-risk neuroblastoma according to the 1993 International Neuroblastoma Response Criteria (INRC) and compare to relevant historical controls.
6 to 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with CR + Very Good Partial Response (VGPR) rate and objective response rate (ORR; CR + VGPR + PR)
Time Frame: 6 to 12 months
Evaluate the combined CR + Very Good Partial Response (VGPR) rate and objective response rate (ORR; CR + VGPR + PR) at EOI per 1993 INRC, compared against historical controls and external controls. Measured by the presence of radiologically assessable disease by cross-sectional computed tomography (CT) or Magnetic resonance imaging (MRI) imaging and/or by metaiodobenzylguanidine (MIBG) or positron emission tomography (PET) scans and bone marrow (BM) response for subjects with newly-diagnosed high-risk neuroblastoma according to the 1993 International Neuroblastoma Response Criteria (INRC).
6 to 12 months
Number of participants with Complete Response (CR) rate per 2017 INRC guidelines
Time Frame: 6 to 12 months
Evaluate CR rate and ORR (CR + PR) at EOI per the 2017 INRC, compared against external controls. Measured by the presence of radiologically assessable disease by cross-sectional computed tomography (CT) or Magnetic resonance imaging (MRI) imaging and/or by metaiodobenzylguanidine (MIBG) or positron emission tomography (PET) scans and bone marrow (BM) response for subjects with newly-diagnosed high-risk neuroblastoma according to the 2017 International Neuroblastoma Response Criteria (INRC).
6 to 12 months
Number of days that subjects remain alive
Time Frame: Through study completion, an average of 1 year, plus 8 years of follow up
Overall survival (OS) for subjects receiving the combination of standard Induction chemotherapy with naxitamab.
Through study completion, an average of 1 year, plus 8 years of follow up
Number of days that subjects remain in remission
Time Frame: Through study completion, an average of 1 year, plus 8 years of follow up
Progression-free survival (PFS) for subjects receiving the combination of standard Induction chemotherapy with naxitamab.
Through study completion, an average of 1 year, plus 8 years of follow up
Number of participants with treatment-related adverse events
Time Frame: At last dose of Naxitamab, average of 6-12 months, plus 42 additional days
At last dose of Naxitamab, average of 6-12 months, plus 42 additional days

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Morphine equivalent daily dose (MEDD) per subject
Time Frame: Year 1
Narcotic use will be quantified for each subject over the course of the study in terms of morphine equivalent daily dose (MEDD).
Year 1

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Giselle Sholler

Collaborators

Y-mAbs Therapeutics

Investigators

  • Study Chair: Jaqueline Kraveka, DO, Medical University of South Carolina

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 14, 2022

Primary Completion (Estimated)

September 1, 2029

Study Completion (Estimated)

September 1, 2036

Study Registration Dates

First Submitted

August 4, 2022

First Submitted That Met QC Criteria

August 4, 2022

First Posted (Actual)

August 5, 2022

Study Record Updates

Last Update Posted (Actual)

February 3, 2026

Last Update Submitted That Met QC Criteria

January 30, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BCC018

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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