An observational study in Neuroblastoma, sponsored by Istituto Giannina Gaslini. Recruiting at 35 sites in Italy. Open to participants aged 0 Days to 31 Years. Per ClinicalTrials.gov, last updated 2026-10-19.
Sponsored by Istituto Giannina Gaslini · Observational
This is an investigator-initiated, multicenter, prospective, non-profit study.
Patients who provide informed consent to participate will be asked to provide a peripheral blood sample, a fresh tumor tissue sample (obtained by biopsy), and/or a bone marrow sample.
All patients with neuroblastoma at diagnosis, with relapsed disease, or with refractory disease may participate, provided that the diagnosis and disease status are established by the treating physician in accordance with national clinical practice and by the participating study center.
Enrolled patients will be reported and registered by means of a paper-based notification form sent by email to the Coordinating Center, which will assign a unique identification code to each patient.
At the time of notification, clinical and treatment-related data will be collected using a paper-based Case Report Form (CRF).
Biological material (peripheral blood, bone marrow, and/or tumor biopsy samples) must be centralized at the Laboratory of Experimental Oncology Therapies, IRCCS Istituto Giannina Gaslini, Genoa, Italy, in accordance with the procedures described in the study protocol. Samples will be processed and analyzed as specified in the protocol.
The results of the initial analysis performed at CEINGE, Naples, will be communicated by the CEINGE Laboratory to the Coordinating Center. A multidisciplinary meeting will then be held with a panel of experts, including oncologists, biologists, pharmacists, and infectious disease specialists. Following this meeting, an individual report will be prepared for each patient.
The report will include the results of Whole Exome Sequencing (WES) or Cancer Gene Panel (CGP) analysis performed on the patient's DNA, together with any potential therapeutic recommendations based on the molecular alterations identified. The report will be sent to the physician responsible for the individual patient.
For patients who provide additional consent by signing the appropriate informed consent form, biological material may also be used for the following purposes:
i) Immunophenotyping, with particular focus on GD2 and B7-H3 expression in tumor biopsy samples and in neuroblastoma cells infiltrating the bone marrow;
ii) High-throughput screening of libraries comprising readily available anticancer drugs;
iii) Generation of in vitro and in vivo tumor models, including primary cell lines and spheroids in vitro, and murine models such as Patient-Derived Xenografts (PDX) and Cell Patient-Derived Xenografts (CPDX) at the Coordinating Center, as well as zebrafish models at San Martino-IST, Genoa, and/or Città della Speranza, Padua.
634 studies on the registry are indexed under Neuroblastoma; 122 are open to participants now.
This study's planned enrollment of 200 is above the median of 121 across 79 observational studies indexed under Neuroblastoma.
Browse Neuroblastoma studies →Istituto Giannina Gaslini is the lead sponsor of 50 studies on the registry; 33 are open to participants now.
Counted across the registry records on this site, refreshed daily.
All patients with newly diagnosed, refractory, or relapsed neuroblastoma referred to AIEOP centers.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Molecular Profiling of Neuroblastoma to Identify Potential Therapeutic Targets
The primary objective of the study is to characterize the molecular profile of neuroblastoma biopsy specimens collected at diagnosis, in cases of first-line treatment refractoriness or disease relapse. Whole-exome sequencing (WES) or comprehensive genomic profiling (CGP) will be performed to identify somatic and/or germline mutations and genetic alterations in individual patients that may represent potential targets for specific drugs.
Time frame: over a 3-year period, starting from the enrollment of the first patient in the study
Identification of Targeted Therapeutic Strategies Based on Somatic Alterations
The secondary objective of the study is, whenever feasible, to suggest a targeted therapeutic strategy within a precision medicine approach, based on the identified somatic alterations.
Time frame: over a 3-year period, starting from the enrollment of the first patient in the study
Started May 2022 (actual). Primary completion is estimated for Dec 2026, 55 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Istituto Giannina Gaslini