Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma
NCT ID: NCT05489887
Last Updated: 2026-01-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
93 participants
INTERVENTIONAL
2022-09-14
2036-09-30
Brief Summary
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Detailed Description
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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
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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HRNB Newly diagnosed subjects
5 cycles of standard of care induction + naxitimab
Naxitimab on Days 1, 3, and 5 of each cycle
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
Interventions
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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 Intervention Names
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Eligibility Criteria
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Inclusion 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
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
12 Months
21 Years
ALL
No
Sponsors
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Y-mAbs Therapeutics
INDUSTRY
Giselle Sholler
OTHER
Responsible Party
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Giselle Sholler
Beat Childhood Cancer Chair
Principal Investigators
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Jaqueline Kraveka, DO
Role: STUDY_CHAIR
Medical University of South Carolina
Locations
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University of Alabama, Children's Alabama
Birmingham, Alabama, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
UCSF Benioff Children's Hospital Oakland-
Oakland, California, United States
Rady Children's Hospital
San Diego, California, United States
Connecticut Children's Hospital
Hartford, Connecticut, United States
University of Florida
Gainesville, Florida, United States
Nicklaus Children's Miami
Miami, Florida, United States
Arnold Palmer Hospital for Children
Orlando, Florida, United States
Augusta University Health
Augusta, Georgia, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States
Kentucky Children's Hospital
Lexington, Kentucky, United States
Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, United States
Cardinal Glennon Children's Hospital
St Louis, Missouri, United States
Levine Children's Hospital
Charlotte, North Carolina, United States
Wake Forest University Health Sciences
Winston-Salem, North Carolina, United States
Randall Children's Hospital
Portland, Oregon, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Dell Children's Blood and Cancer Center
Austin, Texas, United States
Virginia Commonwealth University
Richmond, Virginia, United States
UHC Sainte-Justine
Montreal, Quebec, Canada
CHUQ
Québec, Quebec, Canada
CIUSSS de l'Estrie-CHUS
Sherbrooke, Quebec, Canada
Countries
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Central Contacts
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Facility Contacts
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Related Links
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Beat Childhood Cancer Consortium website
Other Identifiers
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BCC018
Identifier Type: -
Identifier Source: org_study_id
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