Clinical trial · Interventional
Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma
Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients With Non-High-Risk Neuroblastoma
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.
Conditions
Conditions (4)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Ganglioneuroblastoma | Ganglioneuroblastoma | ONTOLOGY_EXACT | 0.90 |
| Localized Resectable Neuroblastoma | Neuroblastoma | CURATED_BROADER | 0.78 |
| Localized Unresectable Neuroblastoma | Neuroblastoma | CURATED_BROADER | 0.78 |
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (11)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Biopsy | Procedure | — | UNRESOLVED |
| Bone Marrow Aspiration | Procedure | — | UNRESOLVED |
| Bone Marrow Biopsy | Procedure | — | UNRESOLVED |
| Carboplatin | Drug | Carboplatin | ALIAS |
| Clinical Observation | Other | — | UNRESOLVED |
| Computed Tomography | Procedure | — | UNRESOLVED |
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Doxorubicin Hydrochloride | Drug | Doxorubicin | ALIAS |
| Etoposide |
Design
Arms and outcomes
Arms (3)
- type
- EXPERIMENTAL
- label
- Group A (clinical observation)
- description
- Patients undergo clinical observation for 96 weeks in the absence of disease progression. Patients also undergo CT, MRI, and/or ultrasound throughout the trial.
- interventionNames
- Other: Clinical Observation
- Procedure: Computed Tomography
- Procedure: Magnetic Resonance Imaging
- Procedure: Ultrasound
- type
- EXPERIMENTAL
- label
- Group B (clinical observation, first-line chemotherapy)
- description
- Patients undergo clinical observation for 3 years in the absence of disease progression. Upon disease progression, patients undergo surgery or receive first-line chemotherapy comprising carboplatin IV over 1 hour on day 1 (courses 1, 2, 4, 6, and 7), etoposide IV over 1 hour on days 1-3 (courses 1, 3, 4, 5, and 7), cyclophosphamide IV over 1 hour on day 1 (courses 2, 3, 5, 6, and 8), and doxorubicin hydrochloride IV over 15 minutes on day 1 (courses 2, 4, 6 and 8). Treatment with chemotherapy repeats every 21 days for 2-8 courses in the absence of disease progression or unacceptable toxicity. Once a PR or better is achieved, patients undergo clinical observation for 3 years. Patients also undergo CT, MRI, and/or ultrasound throughout the trial and undergo bone marrow aspiration, bone marrow biopsy, and tumor biopsy at screening and time of progression.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 18 Months
Show eligibility criteria text
Inclusion Criteria:
* Patients must be:
* \< 12 months (\< 365 days) of age at diagnosis with INRG stage L1; or
* \< 18 months (\< 547 days) of age at diagnosis with INRG stage L2 or stage Ms neuroblastoma/ganglioneuroblastoma
* Enrollment on ANBL00B1 or APEC14B1 is required for all newly diagnosed patients
* Patients must have newly diagnosed v-myc avian myelocytomatosis viral oncogene neuroblastoma derived homolog (MYCN) non-amplified neuroblastoma (International Classification of Diseases for Oncology \[ICD-O\] morphology 9500/3) or MYCN non-amplified ganglioneuroblastoma verified by histology
* Patients must meet the specified criteria for one of the treatment groups defined below; genomic features include MYCN gene amplification, segmental chromosome aberrations (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q) and deoxyribonucleic acid (DNA) index
* "Favorable" genomic features are defined by one or more whole-chromosome gains or hyperdiploid tumor (DNA index \> 1) in the absence of segmental chromosome aberrations as defined above
* "Unfavorable" genomic features are defined by the presence of any segmental chromosome aberration (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q) or diploid tumor (DNA index = 1); this includes copy neutral loss of heterozygosity (LOH)
* Only patients with MYCN non-amplified tumors are eligible for this study
* Group A: patients \< 12 months (\< 365 days) of age with newly diagnosed INRG stage L1 neuroblastoma/ganglioneuroblastoma who meet the following criteria:
* Greatest tumor diameter \< 5 cm of adrenal or non-adrenal origin
* Patients with non-adrenal primaries are eligible, but must have positive uptake on metaiodobenzylguanidine (MIBG) scan or elevated catecholamine metabolites (urine or serum) to support the diagnosis of neuroblastoma
* No prior tumor resection or biopsy
* Group A will be further split into two subsets, which are mutually exclusive, for statistical purposes
* Group A1:
* \> 6 months and \< 12 months of age with an adrenal primary tumor \< 5 cm in greatest diameter OR
* Patients less than 6 months of age with an adrenal primary tumor \> 3.1 and \< 5 cm in greatest diameter OR
* \< 12 months of age with a non-adrenal primary site \< 5 cm in greatest diameter
* Group A2: =\< 6 months of age with an adrenal primary site and tumor =\< 3.1 cm in greatest diameter
* Group B: patients \< 18 months (\< 547 days) of age with newly diagnosed INRG stage L2 neuroblastoma/ganglioneuroblastoma who meet the following criteria:
* No life threatening symptoms or no impending neurologic or other organ function compromise (e.g. epidural or intraspinal tumors with existing or impending neurologic impairment, periorbital or calvarial-based lesions with existing or impending cranial nerve impairment, anatomic or mechanical compromise of critical organ function by tumor \[abdominal compartment syndrome, urinary obstruction, etc.\]); horner syndrome is not considered neurologic compromise
* No prior tumor resection, tumor biopsy ONLY
* Only patients with both favorable histology and favorable genomic features will remain on study as part of Group B; the institution will be notified of histologic and genomic results within 3 weeks of specimen submission on ANBL00B1 or APEC14B1
* Group C: patients \< 18 months (\< 547 days) of age with newly diagnosed INRG stage Ms neuroblastoma/ganglioneuroblastoma
* No prior radiotherapy or chemotherapy, with the exception of dexamethasone, which is allowed
* All patients and/or their parents or legal guardians must sign a written informed consent
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
* Patients with MYCN amplified tumors are not eligible
* Group B and C patients who do not enroll on ANBL1232 within 4 weeks of definitive diagnostic procedure
* Group A and C patients, not required to undergo tumor biopsy, who do not enroll on ANBL1232 within 4 weeks of confirmatory imaging studyReferences
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