Naxitamab Enabled Transplant-Free Study in High-Risk Neuroblastoma (NEXT-NB)

November 5, 2026 updated by: Giselle Sholler

A Beat Childhood Cancer Research Consortium Phase II Trial of Transplant-Free Chemo-immunotherapy Consolidation Regimen With Naxitamab for High-Risk Neuroblastoma

A prospective, multicenter, clinical trial in participants with high-risk neuroblastoma that have completed induction therapy, evaluating the feasibility, efficacy, and toxicity of using anti-GD2 chemo-immunotherapy (naxitamab/irinotecan/temozolomide/GM-CSF) as consolidation followed by standard radiation, then standard anti-GD2 immunotherapy, and 2 years of DFMO maintenance.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

101

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 30 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Age

    1. Participants must be age ≤ 21 years at High-Risk NB diagnosis

    2. Participants must be >12 months of age at enrollment. Diagnosis

    3. Participants must have a pathologically confirmed diagnosis of neuroblastoma and classified as high risk by the criteria used by COG or SIOPEN at the time of diagnosis.

      Exception: patients who are initially diagnosed as non high-risk neuroblastoma, but later converted to high-risk neuroblastoma are also eligible.

      Prior Therapy

    4. Participants must have completed standard induction therapy for initial diagnosis of high-risk neuroblastoma (5-7 cycles of induction chemotherapy with or without anti-GD2 antibody as per section 4.2, may include an additional cycle of therapy given prior to starting HRNB induction), and local control (surgery). Participants will enroll after completion of upfront induction therapy.

      Disease Response

    5. Participants must have a disease response by revised 2017 International Neuroblastoma Response Criteria (INRC) at the end of standard 5-7 cycles of Induction of GEIR:

      o Overall INRC Response ≥PR or

      o Primary tumor ≥SD, AND soft tissue/metastatic bone ≥PR, AND bone marrow ≥MD*

      • Includes bone marrow response of minimal disease or complete response. Timing

    6. Start of therapy to occur ≤ 45 days after completion of last induction cycle (from last day of cycle).

      Organ Function Requirements

    7. Hematological: Hematological recovery as defined by ANC ≥750/μL (unsupported- >24 hrs off G-CSF and 7 days off pegylated filgrastim).

    8. Renal: Participants must have adequate renal function defined:

      o For participants < 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

      o For participants ≥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)

    9. Cardiac: Adequate cardiac function defined as:

      • Shortening fraction of ≥28%, or
      • Ejection fraction of ≥50%

    10. Liver: Adequate liver function defined as:

      • Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
      • ALT (SGPT) < 10 x upper limit of normal (ULN) for age

    11. Post-pubertal study participants 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. Participants who are lactating must agree to stop breast-feeding.

    12. Informed Consent: All participants 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. Participants who are less than 1 year of age 2. Participants that had progressive disease (PD) at any time post cycle 4 during upfront induction therapy.

    3. Participants who required additional therapy after the end of standard Induction (5-7 cycles) to achieve adequate response.

    4. Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.

    5. Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.

    6. Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.

    7. Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Consolidation with anti-GD2 chemo-immunotherapy (naxitamab/irinotecan/temozolomide/GM-CSF)

Consolidation Cycles 1-6: Naxitamab/Irinotecan/Temozolomide/GM-CSF + Radiation Immunotherapy Cycles 1-5: Dinutuximab + GM-CSF + Isotretinoin (or Dinutuximab-Beta) Immunotherapy Cycle 6: Isotretinoin alone DFMO Monotherapy: DFMO for 730 days (2 years)
Drug: Naxitamab and GM-CSF in combination with irinotecan and temozolomide

anti-GD2 chemo-immunotherapy (naxitamab/irinotecan/temozolomide/GM-CSF) as consolidation

Drug: Dinutuximab. Immunotherapy

Dinutuximab + GM-CSF + Isotretinoin (or Dinutuximab-Beta) for 5 cycles plus Isotretinoin alone in Cycle 6

Drug: DFMO

DFMO Monotherapy: DFMO for 730 days (2 years)

Other Names:
  • IWILFIN

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of days that subjects remain in event free
Time Frame: 3 years

To determine whether transplant-free chemo-immunotherapy consolidation (naxitamab/irinotecan/temozolomide/GM-CSF) is non-inferior to the transplant-containing standard with respect to 3-year event-free survival (EFS) from the start of consolidation in participants with high-risk neuroblastoma who have a good end-of-induction response
3 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of days that subjects remain in event free
Time Frame: 5 years

To estimate 5-year EFS from the start of consolidation and compare it descriptively with the 5-year historical transplant-containing benchmark.
5 years
Number of days that subjects remain alive
Time Frame: 5 years

To estimate 3-year and 5-year overall survival (OS) from the start of consolidation and compare it descriptively with the historical transplant-containing benchmark.
5 years
Number of participants with treatment-related adverse events
Time Frame: 5 years

To characterize the morbidity burden of transplant-free consolidation relative to published rates for the transplant-containing standard.
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Giselle Sholler

Collaborators

SERB Pharmaceuticals

Investigators

  • Study Chair: Giselle SaulnierSholler, MD, Penn State Health Golisano Children's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

November 26, 2026

Primary Completion (Estimated)

November 26, 2031

Study Completion (Estimated)

November 26, 2040

Study Registration Dates

First Submitted

November 5, 2026

First Submitted That Met QC Criteria

November 5, 2026

First Posted (Actual)

November 12, 2026

Study Record Updates

Last Update Posted (Actual)

November 12, 2026

Last Update Submitted That Met QC Criteria

November 5, 2026

Last Verified

November 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BCC025

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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