Prospective Study Registry of Peripheral Neuroblastic Tumours Presenting With Spinal Canal Involvement (SCI)

NCT ID: NCT02559804

Last Updated: 2024-04-15

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 150 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2014-05-31
• Study Completion Date: 2029-05-31

Brief Summary

To describe the natural history of peripheral neuroblastic tumour presenting with SCI and evaluate the combined effects of different risk factors on the eventual neurologic and orthopaedic outcomes

Detailed Description

Multi-centre, observational, prospective study registry. About 15% of patients with peripheral neuroblastic tumour present with extradural SCI, of whom approximately 60% are symptomatic. Since SCI may progress to irreversible paraplegia, early diagnosis and prompt treatment is of critical importance.1-3 The treatment options include chemotherapy, neurosurgical decompression and radiation therapy. All may relieve epidural compression, but there is no consensus on which to use first in the individual patient. However, the choice of the treatment could be relevant in the perspective of reducing to the minimum the risk of long-term sequelae.4 Guidelines for the diagnostic work-up and treatment of SCI for International Society of Paediatric Oncology European Neuroblastoma (SIOPEN) neuroblastoma patients were already available in the "guidelines for the treatment of patients with localized resectable neuroblastoma and analysis of prognostic factors" (LNESG1 Protocol), back in 1994,5 although it is unknown how they were applied in the SIOPEN Centres. These guidelines have been reformulated in occasion of the recently activated "European Low and Intermediate risk Neuroblastoma" (LINES Protocol).

There are few publications addressing the diagnosis and treatment of SCI; most are retrospective studies, case reports, or reviews that may be affected by reporting bias.1-9 Therefore new guidelines could be designed based on the information derived from a prospective data collection of newly diagnosed patients.

For this study Spinal Canal Involvement (SCI) is defined when, referring to an axial plane of the spinal cord MRI scan (Figure 1), the tumour extends into the vertebral canal and goes beyond a mentally drown ellipsoid (red circle) passing through the cortical bone of both anterior and posterior arches of the vertebra. This involvement is called "intraspinal" or, better, "intracanal".

Patient data to be collected and included in the corresponding Forms

1. work-up at diagnosis, including radiology report (Computed Tomography or Magnetic Resonance Imaging)

2. treatment administered

3. response to treatment, including radiology report

4. follow-up

5. outcome

Conditions

Neuroblastic Tumors

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

patients NB with SCI

Survival and late effects. Diagnosis of peripheral neuroblastic tumour - peripheral neuroblastic tumour (neuroblastoma, ganglioneuroblastoma, ganglioneuroma) presenting with SCI, symptomatic or asymptomatic, independent of disease extension (stage), and clinical course (first diagnosis or relapse/progression).

survival and late effects

Intervention Type: OTHER

survival and late effects

Interventions

survival and late effects

survival and late effects

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Diagnosis of peripheral neuroblastic tumour - peripheral neuroblastic tumour (neuroblastoma, ganglioneuroblastoma, ganglioneuroma) presenting with SCI, symptomatic or asymptomatic, independent of disease extension (stage), and clinical course (first diagnosis or relapse/progression)
• No previous chemotherapy, except steroids, in the last 6 months
• Age <18 years
• Minimal planned follow-up of 5 years
• Parent/patient written informed consent (Appendix 1(A,B,C))

Exclusion Criteria

• Invasion of intervertebral foramina only

• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Istituto Giannina Gaslini

OTHER

Sponsor Role: collaborator

Associazione Italiana Ematologia Oncologia Pediatrica

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Riccardo Haupt, MD

Role: PRINCIPAL_INVESTIGATOR

Istituto Giannina Gaslini Genova

Locations

Istituto Giannina Gaslini

Genova, , Italy

Site Status: RECRUITING

Countries

Italy

Central Contacts

Riccardo Haupt, MD

Role: CONTACT

riccardohaupt@ospedale-gaslini.ge.it

01056363462 ext. +39

tiziana landi

Role: CONTACT

segreteria@aieop.org

051/2144667 ext. +39

Facility Contacts

Riccardo Haupt, MD

Role: primary

riccardohaupt@ospedale-gaslini.ge.it

01056363462

Stefania Sorrentino

Role: backup

stefaniasorrentino@ospedale-gaslini.ge.it

Other Identifiers

NB with SCI

Identifier Type: -

Identifier Source: org_study_id