Dinutuximab-beta and Chemotherapy in Newly Diagnosed High-Risk Neuroblastoma

NCT07688252 | Not Yet Recruiting Phase 1

• 1 other identifier: TPOG N2020-GD2
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 2

Brief Summary

This clinical trial investigates the safety, side effects, and effectiveness of combining an immunotherapy drug called dinutuximab-beta with standard induction chemotherapy for patients newly diagnosed with high-risk neuroblastoma. Dinutuximab-beta is an antibody designed to target specific molecules on the surface of neuroblastoma cells. In this pilot study, enrolled patients will receive dinutuximab-beta as a continuous intravenous infusion alongside a standard 6-cycle induction chemotherapy regimen. The primary goals of this study are to monitor how well patients tolerate the new combination treatment closely and to determine how effectively tumors shrink before patients proceed to the next phase of their standard consolidation therapy.

Conditions

High Risk Neuroblastoma

Keywords

Neuroblastoma; High-risk; chemoimmumotherapy; newly-diagnosed; dinutuximab-beta

Outcome Measures

Primary Outcomes (2)

• Number of Participants with Unacceptable Toxicities or Toxic Death

  Tolerability is assessed by the number of toxic deaths and the number of patients experiencing unacceptable toxicities. Unacceptable toxicities are assessed according to CTCAE criteria and include: 1. toxicity requiring pressors for ≥ 24 hours (e.g., Grade 4 capillary leak syndrome, Grade 4 anaphylaxis/allergic reaction, or Grade 3-4 hypotension), 2. toxicity requiring ventilatory support for \> 24 hours, 3. Grade 4 or unresolved Grade 3 peripheral motor/sensory neuropathy, 4. Grade 4 acute infusion reaction.

  During Cycles 2-6 of induction therapy (up to approximately 6 months).

• Clinical Response Rate (RR)

  Clinical response rate is defined as the percentage of eligible participants who achieve a Partial Response (PR) or better (e.g., Complete Response or Very Good Partial Response). The tumor response will be evaluated using the revised International Neuroblastoma Response Criteria (INRC).

  At the end of induction therapy (up to approximately 6 months).

Secondary Outcomes (2)

• Event-Free Survival (EFS)

  Up to 10 years from the date of study enrollment.

• Overall Survival (OS)

  Up to 10 years from the date of study enrollment.

Study Arms (1)

Dinutuximab-Beta with Induction Chemotherapy

EXPERIMENTAL

Participants will receive dinutuximab-beta administered as a continuous intravenous infusion in combination with an induction chemotherapy regimen consisting of cyclophosphamide, vincristine, doxorubicin (or epirubicin), etoposide, and cisplatin.

Biological: Dinutuximab Beta; Drug: Induction chemotherapy

Interventions

Dinutuximab Beta BIOLOGICAL

Dinutuximab-beta 10 mg/m²/day administered as a continuous intravenous infusion on Days 0-9 of Cycles 2-6.

Also known as: Qarziba

Dinutuximab-Beta with Induction Chemotherapy

Induction chemotherapy DRUG

Six cycles of standard induction chemotherapy consisting of cyclophosphamide, vincristine, doxorubicin (or epirubicin), etoposide, and cisplatin administered according to the study treatment schedule.

Dinutuximab-Beta with Induction Chemotherapy

Eligibility Criteria

• Age: 1 Year - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Must be 1 to 30 years of age at the time of initial diagnosis.
• Must have histological verification of neuroblastoma or ganglioneuroblastoma, OR demonstration of neuroblastoma cells in the bone marrow with elevated urinary VMA at the time of initial diagnosis.
• Must have newly diagnosed high-risk neuroblastoma according to the revised COG NBL risk classifier (version 2), defined as meeting at least ONE of the following:
• INRG Stage M: MYCN amplification (\> 4-fold increase), OR age 12 to \< 18 months without MYCN amplification but with unfavorable histology/DI = 1/SCA, OR age \> 18 months regardless of biologic features.
• INRG Stage MS: MYCN amplification, OR age 12 to \< 18 months without MYCN amplification but with unfavorable histology/DI = 1/SCA.
• INRG Stage L2: MYCN amplification, OR age 18 months to \< 5 years without MYCN amplification but with unfavorable histology, OR age ≥ 5 years without MYCN amplification but with unfavorable histology (undifferentiated or poorly differentiated tumor).
• INRG Stage L1: Tumor with incomplete resection and MYCN amplification.
• Patients \> 18 months of age initially diagnosed with INRG Stage L1, L2, or MS who are subsequently upgraded to high-risk disease.
• Must have had no prior systemic therapy, or have only completed the first cycle of induction chemotherapy under the TPOG N2020-HR protocol.
• Must have measurable disease, defined as at least ONE of the following (Note: Patients with elevated VMA only are NOT eligible):
• Measurable tumor on MRI or CT scan within 3 weeks prior to study entry (≥ 10 mm in at least one dimension) that is MIBG avid or demonstrates increased FDG/FDOPA uptake on PET scan.
• MIBG or FDG/FDOPA PET scan within 2 weeks prior to study entry with positive uptake at a minimum of one site.
• ECOG Performance Status of 0, 1, or 2.
• Adequate organ function, including:
• Renal: Creatinine clearance or estimated GFR ≥ 60 mL/min/1.73 m², or serum creatinine ≤ upper limit of normal (ULN) based on age/gender.

You may not qualify if:

• Participation in other interventional clinical trials within 1 month.
• Inability to obey the trial instructions.
• Patients with bone marrow failure syndromes.
• Current requirement for immunosuppressive medications (e.g., tacrolimus, cyclosporine, systemic corticosteroids for reasons other than prevention/treatment of acute allergic reactions or adrenal replacement therapy).
• Females who are pregnant or breastfeeding (A pregnancy test is required for female patients of childbearing potential).
• Female patients who are pregnant are ineligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential.
• Lactating females who plan to breastfeed their infants.
• The subject or their partner is planning to conceive
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method during study therapy and for two months after the last dose of (ch14.18/CHO) (dinutuximab-β) are not eligible.

Sponsors & Collaborators

• Chang Gung Memorial Hospital: lead
• National Taiwan University Hospital: collaborator

Study Sites (2)

National Taiwan University Hospital

Taipei, Taiwan, 110, Taiwan

Location

Chang Gung Memorial Hospital Linkou Branch

Taoyuan, Taiwan, 333, Taiwan

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

dinutuximab; Induction Chemotherapy

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

Intervention Hierarchy (Ancestors)

Drug Therapy; Therapeutics; Remission Induction

Study Officials

• Shih-Hsiang Chen, M.D.

  Chang Gung Memorial Hospital

  STUDY CHAIR

• Meng-Yao Lu, M.D.

  National Taiwan University Hospital

  STUDY CHAIR

Central Study Contacts

Shih-Hsiang Chen, M.D.

CONTACT

+886-3-328-1200; samechen@cgmh.org.tw

Meng-Yao Lu, M.D.

CONTACT

+886-2-2312-3456

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: M.D., Pediatric Hematology/Oncology

Study Record Dates

• First Submitted: June 30, 2026
• First Posted: July 7, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): December 31, 2032
• Study Completion (Estimated): December 31, 2032
• Last Updated: July 7, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

TW (2)