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NeuroblastomaPhase 2NCT05489887

Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

A Phase 2 study testing Naxitamab in people with Neuroblastoma, run by Giselle Sholler at 26 sites in 4 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 12 months to 21 years
Conditions the study is for
Neuroblastoma
What is being tested?
Naxitamab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
93 participants
Where
26 sitesin 4 countries
Study dates
14 Sep 2022to Sep 2036
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 months to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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:
  • Subjects with newly diagnosed neuroblastoma with INRGSS Stage M disease with either of the following features:
  • MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
  • 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
  • Age > 547 days of age regardless of biologic features
  • Subjects with newly diagnosed neuroblastoma with INRGSS Stage MS disease with either of the following:
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  • MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals); OR
  • 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:
  • MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals); OR
  • 18 months to <5 years of age without MYCN amplification, but with unfavorable histology (INPC); OR
  • ≥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.
  • Subjects must be age ≤ 21 years at initial diagnosis.
  • Subjects must be >12 months of age at enrollment.
  • Adequate cardiac function defined as:
  • Shortening fraction of ≥ 27% by echocardiogram, or
  • Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.
  • Adequate liver function must be demonstrated, defined as:
  • Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
  • ALT (SGPT) < 5 x upper limit of normal (ULN) for age
  • 1. Subjects must have adequate renal function defined as:
  • For subjects < 17 years old: 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 mL/min/1.73 m2. The Bedside Schwartz equation is: [(0.413) X (Height in cm)] / SCr
  • For subjects ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: [(140-age) x (Wt in kg) x (0.85 if female)] / (72 x SCr)
  • OR a 24 hour urine Creatinine clearance ≥ 70 mL/min/1.73 m2
  • A negative serum pregnancy test is required for subjects of childbearing potential (≥13 years of age or after onset of menses)
  • 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 (and assent, as applicable) 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.
  • Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines and applicable local regulations.

Who cannot join

  • Subjects who are less than 1 year of age
  • 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.
  • 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.
  • Treatment with immunosuppressive treatment (topical, inhaled and short-term emergency steroids excluded) within 4 weeks prior to enrollment
  • 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.
  • Pregnant or breastfeeding (NOTE: breast milk cannot be stored for future use while the mother is being treated on study.)
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  • Subjects receiving any investigational drug concurrently.
  • 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
  • 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.

Trial locations and contacts

26 sites in 4 countries. Contact the study team or a site near you to ask about taking part.

BCC Enroll contact

7175310003

BCCEnroll@pennstatehealth.psu.edu

26 sites in 4 countries

United States21 sites, 20 recruiting

  • Augusta University Health

    Augusta, Georgia, 30912

    Kimberly Gray, kigray@augusta.edu

    Coleen McDonough

    Recruiting

  • Dell Children's Blood and Cancer Center

    Austin, Texas, 78723

    Rhea Robinson, rmrobinson@ascension.org

    Virginia Harrod

    Recruiting

  • University of Alabama/Children's of Alabama

    Birmingham, Alabama, 35201

    Bridget Tate, btate@peds.uab.edu

    Elizabeth Alva

    Recruiting

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

    Shanta Salzar, salzers@musc.edu

    Jaqueline Kraveka

    Recruiting

  • Levine Children's Hospital

    Charlotte, North Carolina, 28204

    Jontyce Green, Jontyce.Green@advocatehealth.org

    Thomas Russell

    Recruiting

  • Duke University

    Durham, North Carolina, 27708

    Morgan Low, morgan.low@duke.edu

    Jessica Sun

    Recruiting

  • University of Florida

    Gainesville, Florida, 32611

    Ashley Bayne, abayne@UFL.EDU

    Joanne Lagmay

    Recruiting

  • Connecticut Children's Hospital

    Hartford, Connecticut, 06106

    Adam Barselau, Abarselau@connecticutchildrens.org

    Michael Isakoff

    Recruiting

  • Penn State Milton S. Hershey Medical Center and Children's Hospital

    Hershey, Pennsylvania, 17033

    Penn State Clinical Trials, ExtractClinicalTrials@pennstatehealth.psu.edu

    Lisa McGregor

    Recruiting

  • Kapiolani Medical Center for Women and Children

    Honolulu, Hawaii, 96813

    Andrea Siu, andrea.siu@kapiolani.org

    Kelley Hutchins

    Recruiting

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

    Susan Hall, HallSF@archildrens.org

    Kevin Bielamowicz

    Recruiting

  • Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine

    Louisville, Kentucky, 40202

    Jennifer Miller, Jennifer.Miller4@nortonhealthcare.org

    Michael Ferguson

    Recruiting

  • Nicklaus Children's Hospital

    Miami, Florida, 33155

    Aixa Guadarrama, Aixa.Guadarrama@Nicklaushealth.org

    Maggie Fader

    Recruiting

  • Children's Hospital and Clinics of Minnesota

    Minneapolis, Minnesota, 55404

    Pauline Mitby, pauline.mitby@childrensmn.org

    Jawhar Rawwas

    Recruiting

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609

    Group Contact, PedOncRschOAK@ucsf.edu

    Jennifer Michlitsch

    Recruiting

  • Arnold Palmer Hospital for Children

    Orlando, Florida, 32806

    Marie Frankos, marie.frankos@orlandohealth.com

    Jaime Libes-Bander

    Recruiting

  • Randall Children's Hospital

    Portland, Oregon, 97227

    Aaron White, AJWHITE@lhs.org

    Jason Glover

    Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia, 23284

    Mary Madu, memadu@vcu.edu

    Madhu Gowda

    Recruiting

  • Rady Children's Hospital

    San Diego, California, 92123

    Megan Saenz, msaenz@rchsd.org

    William Roberts

    Recruiting

  • Cardinal Glennon Children's Hospital

    St Louis, Missouri, 63104

    Gina Martin, gina.martin@health.slu.edu

    William Ferguson

    Recruiting

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157

    Withdrawn

Other trials in United States

Canada3 sites, 3 recruiting

  • UHC Sainte-Justine

    Montreal, Quebec, QC H3S 2G4

    Guillaume Leblanc, guillaume.leblanc.hsj@ssss.gouv.qc.ca

    Pierre Tiera

    Recruiting

  • CHUQ

    Québec, Quebec, QC G1V 4W6

    Valérie-Ève Julien, Valerie-Eve.Julien@crchudequebec.ulaval.ca

    Bruno Michon

    Recruiting

  • CIUSSS de l'Estrie-CHUS

    Sherbrooke, Quebec, QC J1H 5H3

    Cassandra Leblanc Desrochers, cassandra.leblanc-desrochers.ciussse-chus@ssss.gouv.qc.ca

    Josee Brossard

    Recruiting

Other trials in Canada

Brazil1 site, 1 recruiting

  • GRAACC Hospital

    São Paulo, São Paulo, 04039-001

    GRAACC Group Email, pesquisaclinica@graacc.org.br

    Eliana Maria Monteiro Caran, Dra

    Recruiting

Other trials in Brazil

Spain1 site, 1 recruiting

  • Hospital Sant Joan de Déu

    Esplugues de Llobregat, Barcelona, 08950

    Soraya Peralta Garzon, soraya.peralta@sjd.es

    Jaume Mora

    Recruiting

Other trials in Spain

About this study

Official title: 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.

Read the 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.

Conditions
Neuroblastoma
Being tested
Naxitamab
Keywords
naxitimab, induction
Medical subject headings
Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NaxitamabDrug

    Also called Danyelza

    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

Study groups in NCT05489887
Study groupRoleWhat they receive
HRNB Newly diagnosed subjectsExperimental5 cycles of standard of care induction + naxitimab Naxitimab on Days 1, 3, and 5 of each cycle
Receives: Drug: Naxitamab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with Complete Response (CR) rate per 1993 INRC guidelines

    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.

    Measured: 6 to 12 months

Secondary outcomes (5)

  1. Number of participants with CR + Very Good Partial Response (VGPR) rate and objective response rate (ORR; CR + VGPR + PR)

    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).

    Measured: 6 to 12 months

  2. Number of participants with Complete Response (CR) rate per 2017 INRC guidelines

    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).

    Measured: 6 to 12 months

  3. Number of days that subjects remain alive

    Overall survival (OS) for subjects receiving the combination of standard Induction chemotherapy with naxitamab.

    Measured: Through study completion, an average of 1 year, plus 8 years of follow up

  4. Number of days that subjects remain in remission

    Progression-free survival (PFS) for subjects receiving the combination of standard Induction chemotherapy with naxitamab.

    Measured: Through study completion, an average of 1 year, plus 8 years of follow up

  5. Number of participants with treatment-related adverse events

    Measured: At last dose of Naxitamab, average of 6-12 months, plus 42 additional days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Aug 2022
  2. Study start14 Sep 2022
  3. Primary completionSep 2029 (estimated)
  4. Study completionSep 2036 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
93 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05489887
Sponsor's protocol ID
BCC018
Status last verified
Aug 2026
First submitted
4 Aug 2022
Last updated
12 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Naxitamab in people with Neuroblastoma, run by Giselle Sholler at 26 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 29 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 12 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 26 sites in United States, Canada, Brazil, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact BCC Enroll (7175310003, BCCEnroll@pennstatehealth.psu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with Complete Response (CR) rate per 1993 INRC guidelines (time frame: 6 to 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05489887.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2026.

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