Neuroblastic TumorsNB-SCINCT02559804
Prospective Study Registry of Peripheral Neuroblastic Tumours Presenting With Spinal Canal Involvement (SCI)
An observational study of Neuroblastic Tumors, run by Associazione Italiana Ematologia Oncologia Pediatrica at 1 site in Italy.
Trial 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 Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Neuroblastic Tumors
- What is being tested?
- survival and late effects
- How many people
- 150 participants
- Where
- 1 siteItaly
- Study dates
- May 2014to May 2029
- How long will I take part?
- 5 Years
- 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients in the age range 0-18 years diagnosed with peripheral neuroblastic tumour and SCI in the period from May 2014 to April 2017
Who may be able to join
- 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))
Who cannot join
- Invasion of intervertebral foramina only
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
Istituto Giannina Gaslini
Genova, 16147
Riccardo Haupt, MD, 01056363462, riccardohaupt@ospedale-gaslini.ge.it
Stefania Sorrentino, stefaniasorrentino@ospedale-gaslini.ge.it
About this study
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
Read the 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
- work-up at diagnosis, including radiology report (Computed Tomography or Magnetic Resonance Imaging)
- treatment administered
- response to treatment, including radiology report
- follow-up
- outcome
- Conditions
- Neuroblastic Tumors
- Being tested
- survival and late effects
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
survival and late effectsOther
survival and late effects
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Other: survival and late effects |
What is being measured
The primary outcome is the main question the study is designed to answer.
prevalence of neurologic consequences
American Spinal Injury Association ASIA impairment scale
Measured: for 5 years
prevalence of orthopedic consequences
using Common Terminology Criteria for Adverse Events v. 4.0
Measured: for 5 years
prevalence of pain
using Face, Legs, Activity, Cry, Consolability scale (FLACC scale)
Measured: for 5 years
Secondary outcomes (2)
relapse or second tumour
event free survival
Measured: 5 years
survival
overall survival
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2015 (estimated)
- Study startMay 2014
- Primary completionMay 2029 (estimated)
- Study completionMay 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuroblastic TumorsStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 150 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Associazione Italiana Ematologia Oncologia Pediatrica
- Sponsor type
- University, hospital or other
- Collaborators
- Istituto Giannina Gaslini
- Responsible party
- Sponsor
- Investigators
- Riccardo Haupt, MD, Principal investigator, Istituto Giannina Gaslini Genova
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02559804
- Sponsor's protocol ID
- NB with SCI
- Acronym
- NB-SCI
- Status last verified
- Apr 2024
- First submitted
- 11 Sep 2015
- Last updated
- 15 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Study Registry of Peripheral Neuroblastic Tumours Presenting With Spinal Canal Involvement (SCI)
- ClinicalTrials.gov ID
- NCT02559804
- Acronym / sponsor ID
- NB-SCI / NB with SCI
- Conditions
- Neuroblastic Tumors
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Associazione Italiana Ematologia Oncologia PediatricaUniversity, hospital or other
- Collaborators
- Istituto Giannina Gaslini
- Study start
- May 2014
- Primary completion
- May 2029 (estimated)
- Study completion
- May 2029 (estimated)
- First posted
- 24 Sep 2015 (estimated)
- 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.
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
- work-up at diagnosis, including radiology report (Computed Tomography or Magnetic Resonance Imaging)
- treatment administered
- response to treatment, including radiology report
- follow-up
- outcome
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients NB with SCI | – | Other: survival and late effectsSurvival 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). |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients in the age range 0-18 years diagnosed with peripheral neuroblastic tumour and SCI in the period from May 2014 to April 2017
Key inclusion criteria (5 listed)
- 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))
Key exclusion criteria (1 listed)
- Invasion of intervertebral foramina only
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| survival and late effectssurvival and late effects | Other | patients NB with SCI |
Primary outcome
prevalence of neurologic consequences
Time frame: for 5 years
American Spinal Injury Association ASIA impairment scale
prevalence of orthopedic consequences
Time frame: for 5 years
using Common Terminology Criteria for Adverse Events v. 4.0
prevalence of pain
Time frame: for 5 years
using Face, Legs, Activity, Cry, Consolability scale (FLACC scale)
2 secondary outcomes
relapse or second tumour
Time frame: 5 years
survival
Time frame: 5 years
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 15 Apr 2024.
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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.
Riccardo Haupt, MD principal investigator
Istituto Giannina Gaslini Genova
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuroblastic Tumors, run by Associazione Italiana Ematologia Oncologia Pediatrica at 1 site 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 1 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 15 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Contact Riccardo Haupt, MD (01056363462 ext. +39, riccardohaupt@ospedale-gaslini.ge.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
prevalence of neurologic consequences (time frame: for 5 years). The study lists 3 primary outcomes in all. 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 15 Apr 2024.