Promising antibody added to standard chemo for tough childhood cancer

NCT ID NCT05489887

Disease control Sponsor: Giselle Sholler Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 4 times

Summary

This phase II trial is testing whether adding the drug naxitamab (Danyelza) to standard chemotherapy improves outcomes for children newly diagnosed with high-risk neuroblastoma. About 93 participants will receive naxitamab during five cycles of induction chemo. The goal is to see if more children achieve a complete response and have better survival compared to past treatments.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Naxitamab (Danyelza), a monoclonal antibody that targets GD2 on cancer cells
What this could lead to: If successful, adding naxitamab early in treatment could improve response rates and survival for children with high-risk neuroblastoma.
What could go wrong: This is a phase II trial with a modest size (93 participants) and no control group—results are compared to historical data. Side effects from naxitamab, like pain and allergic reactions, are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 93 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2022

Expected to finish

Sep 2036

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 months to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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. 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 8. A negative serum pregnancy test is required for subjects 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 (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. 10. 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. 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.

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Conditions

The condition(s) this trial relates to.

neuroblastoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

High-risk neuroblastoma Neuroblastoma

How to take part

Only the study team decides who joins. These are the ways to reach them.

The study's own enquiry address

This study publishes an address for enquiries. See it below .
The places running it

25 sites in 4 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

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A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

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Locations

Arkansas Children's Hospital
RECRUITING

Little Rock, Arkansas, 72202, United States
Arnold Palmer Hospital for Children
RECRUITING

Orlando, Florida, 32806, United States
Augusta University Health
RECRUITING

Augusta, Georgia, 30912, United States
CHUQ
RECRUITING

Québec, Quebec, QC G1V 4W6, Canada
CIUSSS de l'Estrie-CHUS
RECRUITING

Sherbrooke, Quebec, QC J1H 5H3, Canada
Cardinal Glennon Children's Hospital
RECRUITING

St Louis, Missouri, 63104, United States
Children's Hospital and Clinics of Minnesota
RECRUITING

Minneapolis, Minnesota, 55404, United States
Connecticut Children's Hospital
RECRUITING

Hartford, Connecticut, 06106, United States
Dell Children's Blood and Cancer Center
RECRUITING

Austin, Texas, 78723, United States
Duke University
RECRUITING

Durham, North Carolina, 27708, United States
GRAACC Hospital
RECRUITING

São Paulo, São Paulo, 04039-001, Brazil

Contact Email: •••••@•••••
Hospital Sant Joan de Déu
RECRUITING

Esplugues de Llobregat, Barcelona, 08950, Spain
Kapiolani Medical Center for Women and Children
RECRUITING

Honolulu, Hawaii, 96813, United States
Levine Children's Hospital
RECRUITING

Charlotte, North Carolina, 28204, United States
Medical University of South Carolina
RECRUITING

Charleston, South Carolina, 29425, United States
Nicklaus Children's Hospital
RECRUITING

Miami, Florida, 33155, United States
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine
RECRUITING

Louisville, Kentucky, 40202, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
RECRUITING

Hershey, Pennsylvania, 17033, United States
Rady Children's Hospital
RECRUITING

San Diego, California, 92123, United States
Randall Children's Hospital
RECRUITING

Portland, Oregon, 97227, United States
UCSF Benioff Children's Hospital Oakland
RECRUITING

Oakland, California, 94609, United States
UHC Sainte-Justine
RECRUITING

Montreal, Quebec, QC H3S 2G4, Canada
University of Alabama/Children's of Alabama
RECRUITING

Birmingham, Alabama, 35201, United States
University of Florida
RECRUITING

Gainesville, Florida, 32611, United States
Virginia Commonwealth University
RECRUITING

Richmond, Virginia, 23284, United States
Wake Forest University Health Sciences
WITHDRAWN

Winston-Salem, North Carolina, 27157, United States

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