Neuroblastoma Clinical Trials

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.