Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

Recruiting now · Phase 2

Conditions studied: Neuroblastoma

In brief

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.

Key facts

Study ID

NCT05489887

Run by

Giselle Sholler

People needed

93

Starts

2022-09-14

Expected to finish

2036-09-01

Last updated by the study team

2026-07-01

Who can join

Age: 1 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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:
• 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)

You may not qualify if…

• 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.)
• 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.

Where it is running

• Hospital Sant Joan de Déu — Esplugues de Llobregat, Barcelona, Spain (enrolling)
• Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States (enrolling)
• Rady Children's Hospital — San Diego, California, United States (enrolling)
• Connecticut Children's Hospital — Hartford, Connecticut, United States (enrolling)
• University of Florida — Gainesville, Florida, United States (enrolling)
• Nicklaus Children's Hospital — Miami, Florida, United States (enrolling)
• Arnold Palmer Hospital for Children — Orlando, Florida, United States (enrolling)
• Augusta University Health — Augusta, Georgia, United States (enrolling)
• Kapiolani Medical Center for Women and Children — Honolulu, Hawaii, United States (enrolling)
• Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine — Louisville, Kentucky, United States (enrolling)
• Children's Hospital and Clinics of Minnesota — Minneapolis, Minnesota, United States (enrolling)
• Cardinal Glennon Children's Hospital — St Louis, Missouri, United States (enrolling)
• Levine Children's Hospital — Charlotte, North Carolina, United States (enrolling)
• University of Alabama/Children's of Alabama — Birmingham, Alabama, United States (enrolling)
• Randall Children's Hospital — Portland, Oregon, United States (enrolling)
• Penn State Milton S. Hershey Medical Center and Children's Hospital — Hershey, Pennsylvania, United States (enrolling)
• Medical University of South Carolina — Charleston, South Carolina, United States (enrolling)
• Dell Children's Blood and Cancer Center — Austin, Texas, United States (enrolling)
• Virginia Commonwealth University — Richmond, Virginia, United States (enrolling)
• GRAACC Hospital — São Paulo, Brazil (enrolling)
• UHC Sainte-Justine — Montreal, Quebec, Canada (enrolling)
• CHUQ — Québec, Quebec, Canada (enrolling)
• CIUSSS de l'Estrie-CHUS — Sherbrooke, Quebec, Canada (enrolling)
• Wake Forest University Health Sciences — Winston-Salem, North Carolina, United States

Full record on ClinicalTrials.gov

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