NeuroblastomaPREMENCT07881172
Prospective Multicenter Study of the Genomic, Transcriptomic, and Biological Profile of Patients With Neuroblastoma
An observational study of Neuroblastoma, run by Istituto Giannina Gaslini at 35 sites in Italy.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 0 days to 31 years
- Conditions the study is for
- Neuroblastoma
- How many people
- 200 participants
- Where
- 35 sitesItaly
- Study dates
- 12 May 2022to 31 Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 days to 31 years
- All sexes
- Child, Adult
Study population: All patients with newly diagnosed, refractory, or relapsed neuroblastoma referred to AIEOP centers.
Who may be able to join
- All patients diagnosed with neuroblastoma at AIEOP centers.
- Signed informed consent/assent provided by the subject and/or their parents or legal guardians.
- Age 0 to ≤ 31 years.
- Life expectancy > 3 months.
- Availability of fresh tumor tissue and/or bone marrow and peripheral blood samples.
Who cannot join
- Absence of signed informed consent/assent from the subject and/or their parents or legal guardians.
- Life expectancy < 3 months.
Trial locations and contacts
35 sites in 1 country. Contact the study team or a site near you to ask about taking part.
35 sites in 1 country
Italy35 sites, 35 recruiting
- Recruiting
Azienda Ospedali Riuniti Presidio "G. Salesi", SOS Oncoematologia pediatrica
Ancona
Paolo Pierani, +39 071 5962130, paolo.pierani@ospedaliriuniti.marche.it
- Recruiting
AO Policlinico, Dipartimento di pediatria
Bari
Francesco De Leonardis, +39 080 559 1111, prenotazioni@policlinico.ba.it
- Recruiting
Ospedale Papa Giovanni XXIII USS Oncoematologia Pediatric
Bergamo
Massimo Provenzi, +39035/2673239, mprovenzi@hpg23.it
- Recruiting
Oncologia ed Ematologia Pediatrica "Lalla Seragnoli" Clinica Pediatrica Policlinico Sant'Orsola Malpighi
Bologna
Fraia Melchionda, +39 051 214 4443, fraia.melchionda@aosp.bo.it
- Recruiting
Spedali civili, presidio Ospedale dei Bambini oncoematologia pediatrica e TMO
Brescia
Carmelita D'Ippolito, +39030 399.5711, oncoematologia.trapiantomidollo.pediatrico@asst-spedalicivili.it
- Recruiting
SC Oncoematologia Pediatrica e Patologia della Coagulazione, Ospedale Pediatrico Microcitemico "Antonio Cao", Azienda Ospedaliera Brotzu
Cagliari
Rosa Maria Mura, +39 070 6095686, rosamaria.mura@aob.it
- Recruiting
AOU Policlinico Vittorio Emanuele, UOC Ematologia ed oncologia pediatrica con TMO
Catania
Andrea Di Cataldo, +39095 3782429, adicata@unict.it
- Recruiting
AO Pugliese-Ciaccio, UOC di Ematologia ed Oncologia Pediatric
Catanzaro
Eulalia Galea, +39 0961 883111, NA@NA.NA
- Recruiting
SO "Annunziata" UOC Pediatria
Cosenza
Domenico Sperlì, +390984-681342, d.sperli@aocs.it
- Recruiting
AOU Sant'Anna di Ferrara, SSD Oncoematologia Pediatric
Ferrara
Roberta Burnelli, +390532 239068, roberta.burnelli@unife.it
- Recruiting
A.O.U Meyer Centro di Eccellenza Oncologia ed Ematologia pediatrica - SOC Oncologia, Ematologia e Trapianto di Cellule Staminali Emopoietiche
Florence
Annalisa Tondo, +39055 56622739, annalisa.tondo@meyer.it
- Recruiting
IRCCS Istituto Giannina Gaslini
Genova
Massimo Conte, +39 010 5636 2410, massimoconte@gaslini.org
- Recruiting
Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico
Milan
Anna Maria Fagnani, +39 02-5460394, annamaria.fagnani@policlinico.mi.it
- Recruiting
Fondazione IRCCS Istituto Nazionale Tumori SC Pediatria Oncologica
Milan
Roberto Luksch, +39 02 23902588, roberto.luksch@istitutotumori.mi.it
- Recruiting
Azienda Policlinico di Modena UO Pediatria ad indirizzo oncoematologico
Modena
Giovanni Palazzi, +39059 4222652, palazzi.giovanni@aou.mo.it
- Recruiting
AORN Santobono-Pausilipon, Dipartimento di Oncoematologia
Naples
Maria Rosaria Augurio, +39081 2205111, NA@NA.NA
- Recruiting
Università degli Studi della Campania "Luigi Vanvitelli" UOSD Ematologie e Oncologia Pediatrica DAI Materno infantile
Naples
Elvira Pota, +39 081 566 5438, dip.donnabambinoechirurgia@unicampania.it
- Recruiting
Azienda Ospedaliera di Padova. Oncoematologia pediatrica
Padova
Elisabetta Viscardi, +39049 821 3579, elisabetta.viscardi@unipd.it
- Recruiting
ARNAS Civico di Cristina e Benfratelli UOC Oncoematologia Pediatrica
Palermo
Paolo D'Angelo, +39091 6664143, oncoematoped@arnascivico.it
- Recruiting
Azienda Ospedaliero Universitaria di Parma, UOC di Pediatria ed Oncoematologia
Parma
Patrizia Bertolini, +390521 702831, pbertolini@ao.pr.it
- Recruiting
Fondazione Irccs, Policlinico San Matteo, SC Oncoematologia pediatrica
Pavia
Alessandra Tolva, +390382.502067, a.tolva@smatteo.pv.it
- Recruiting
Ospedale 'S.M. della Misericordia'. A.O. Perugia S.C. di Oncoematologia Pediatrica con Trapianto di CSE
Perugia
Katia Perruccio, +39075 5781, katia.perruccio@ospedale.perugia.it
- Recruiting
Ospedale Civile dello Spirito Santo, Diaprtimento di ematologia, medicina trasfusionale e biotecnologie
Pescara
Daniela Onofrillo, +39085 4252741, daniela.onofrillo@asl.pe.it
- Recruiting
Ospedale S. Chiara, AOU Pisana, UO Oncoematologia pediatrica
Pisa
Gabriella Casazza, +39050-992841, g.casazza@ao-pisa.toscana.it
- Recruiting
AO "Bianchi Melacrino Morelli", UOC Ematologia
Reggio Calabria
Francesca Ronco, +390965 397195, NA@NA.NA
- Recruiting
Ospedale Infermi U.O. di Pediatria SS Oncoematologia pediatrica
Rimini
Roberta Pericoli, +390541 653181, roberta.pericoli@auslromagna.it
- Recruiting
IRCCS Ospedale Pediatrico Bambino Gesu, Dip.to Ematologia Oncologia e medicina trasfusionale
Roma
Maria Antonietta De Ioris, +39 06 6859 4664, mantoniettadeioris@opbg.net
- Recruiting
Policlinico Gemelli, UOC Oncologia Pediatrica
Roma
Stefano Mastrangelo, +3906 3015.1, stefano.mastrangelo@policlinicogemelli.it
- Recruiting
Policlinico Umberto I, Universita' "la Sapienza" UOC Oncoematologia pediatrica
Roma
Amalia Schiavetti, +3906 49979348, amalia.schiavetti@uniroma1.it
- Recruiting
IRCCS Ospedale "Casa Sollievo Della Sofferenza", UOC Oncoematologia pediatrica
San Giovanni Rotondo
Anita Spirito, +390882 410111, a.spirito@operapadrepio.it
- Recruiting
AOU Sassari, Clinica Pediatrica
Sassari
Nadia Vacca, +39079 228237, nadia.vacca@aousassari.it
- Recruiting
Policlinico "Le Scotte", clinica pediatrica
Siena
Daniela Galimberti, +390577 586513, d.galimberti@ao-siena.toscana.it
- Recruiting
AOU Citta' della Salute e della scienza di Torino, Presidio Infantile Regina Margherita, SC Oncoematologia Pediatrica e centro Trapianti
Torino
Elisa Tirtei, +39011 3135258, etirtei@cittadellasalute.to.it
- Recruiting
IRCCS Materno Infantile "Burlo Garofolo" , Dipartimento pediatrico SC Oncoematologia pediatrica ed SS Trapianto di midollo
Trieste
Marco Rabusin, +39040.3785.563, marco.rabusin@burlo.trieste.it
- Recruiting
Policlinico 'G.B. Rossi - AOUI Verona, U.O.C Oncoematologia Pediatrica
Verona
Simone Cesaro, +39 045 812 7874, simone.cesaro@aovr.veneto
About this study
Official title: Protocollo Multicentrico Prospettico Studio Del Profilo Genomico, Trascrittomico e Biologico in Pazienti Affetti da Neuroblastoma / Prospective Multicenter Study of the Genomic, Transcriptomic, and Biological Profile of Patients With Neuroblastoma
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.
Read more
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:
- 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.
- Conditions
- Neuroblastoma
- Medical subject headings
- Neuroblastoma
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: over a 3-year period, starting from the enrollment of the first patient in the study
Secondary outcomes (1)
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.
Measured: over a 3-year period, starting from the enrollment of the first patient in the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2026
- Study start12 May 2022
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Istituto Giannina Gaslini
- Sponsor type
- University, hospital or other
- Collaborators
- Associazione Italiana Ematologia Oncologia Pediatrica
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07881172
- Sponsor's protocol ID
- PREME (12186)
- Acronym
- PREME
- Status last verified
- Oct 2026
- First submitted
- 12 Oct 2026
- Last updated
- 19 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Protocollo Multicentrico Prospettico Studio Del Profilo Genomico, Trascrittomico e Biologico in Pazienti Affetti da Neuroblastoma / Prospective Multicenter Study of the Genomic, Transcriptomic, and Biological Profile of Patients With Neuroblastoma
- ClinicalTrials.gov ID
- NCT07881172
- Acronym / sponsor ID
- PREME / PREME (12186)
- Conditions
- Neuroblastoma
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Istituto Giannina GasliniUniversity, hospital or other
- Collaborators
- Associazione Italiana Ematologia Oncologia Pediatrica
- Study start
- 12 May 2022 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 19 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 0 days to 31 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- All patients with newly diagnosed, refractory, or relapsed neuroblastoma referred to AIEOP centers.
Key inclusion criteria (5 listed)
- All patients diagnosed with neuroblastoma at AIEOP centers.
- Signed informed consent/assent provided by the subject and/or their parents or legal guardians.
- Age 0 to ≤ 31 years.
- Life expectancy > 3 months.
- Availability of fresh tumor tissue and/or bone marrow and peripheral blood samples.
Key exclusion criteria (2 listed)
- Absence of signed informed consent/assent from the subject and/or their parents or legal guardians.
- Life expectancy < 3 months.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Molecular Profiling of Neuroblastoma to Identify Potential Therapeutic Targets
Time frame: over a 3-year period, starting from the enrollment of the first patient in the study
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.
1 secondary outcome
Identification of Targeted Therapeutic Strategies Based on Somatic Alterations
Time frame: over a 3-year period, starting from the enrollment of the first patient in the study
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Azienda Ospedali Riuniti Presidio "G. Salesi", SOS Oncoematologia pediatrica
Ancona, Italy
Ospedale Papa Giovanni XXIII USS Oncoematologia Pediatric
Bergamo, Italy
35 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuroblastoma, run by Istituto Giannina Gaslini at 35 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Molecular Profiling of Neuroblastoma to Identify Potential Therapeutic Targets (time frame: over a 3-year period, starting from the enrollment of the first patient in the study). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026.