Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

NCT05489887 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: BCC018
• Study Type: interventional
• Target: 93
• Locations: 2 countries
• Sites: 23

Brief 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. 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.

Conditions

Neuroblastoma

Keywords

naxitimab; induction

Outcome Measures

Primary Outcomes (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.

  6 to 12 months

Secondary Outcomes (5)

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

  6 to 12 months

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

  6 to 12 months

• Number of days that subjects remain alive

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

• Number of days that subjects remain in remission

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

• Number of participants with treatment-related adverse events

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

Study Arms (1)

HRNB Newly diagnosed subjects

EXPERIMENTAL

5 cycles of standard of care induction + naxitimab Naxitimab on Days 1, 3, and 5 of each cycle

Drug: Naxitamab

Interventions

Naxitamab DRUG

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

Also known as: Danyelza

HRNB Newly diagnosed subjects

Eligibility Criteria

• Age: 12 Months - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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
• 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
• 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
• 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.

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

Sponsors & Collaborators

• Giselle Sholler: lead
• Y-mAbs Therapeutics: collaborator

Study Sites (23)

University of Alabama/Children's of Alabama

Birmingham, Alabama, 35201, United States

RECRUITING

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

RECRUITING

Rady Children's Hospital

San Diego, California, 92123, United States

RECRUITING

Connecticut Children's Hospital

Hartford, Connecticut, 06106, United States

RECRUITING

University of Florida

Gainesville, Florida, 32611, United States

RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

RECRUITING

Augusta University Health

Augusta, Georgia, 30912, United States

RECRUITING

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96813, United States

RECRUITING

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

Louisville, Kentucky, 40202, United States

RECRUITING

Children's Hospital and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

RECRUITING

Cardinal Glennon Children's Hospital

St Louis, Missouri, 63104, United States

RECRUITING

Levine Children's Hospital

Charlotte, North Carolina, 28204, United States

RECRUITING

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

WITHDRAWN

Randall Children's Hospital

Portland, Oregon, 97227, United States

RECRUITING

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

Hershey, Pennsylvania, 17033, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

Dell Children's Blood and Cancer Center

Austin, Texas, 78723, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23284, United States

RECRUITING

UHC Sainte-Justine

Montreal, Quebec, QC H3S 2G4, Canada

RECRUITING

CHUQ

Québec, Quebec, QC G1V 4W6, Canada

RECRUITING

CIUSSS de l'Estrie-CHUS

Sherbrooke, Quebec, QC J1H 5H3, Canada

RECRUITING

Related Links

• Beat Childhood Cancer Consortium website

MeSH Terms

Conditions

Neuroblastoma

Interventions

naxitamab

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Jaqueline Kraveka, DO

  Medical University of South Carolina

  STUDY CHAIR

Central Study Contacts

BCC Enroll

CONTACT

7175310003; BCCEnroll@pennstatehealth.psu.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Beat Childhood Cancer Chair

Study Record Dates

• First Submitted: August 4, 2022
• First Posted: August 5, 2022
• Study Start: September 14, 2022
• Primary Completion (Estimated): September 1, 2029
• Study Completion (Estimated): September 1, 2036
• Last Updated: February 3, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3); US (20)