RetinoblastomaNCT02329002
SPT Screening in Irradiated Hereditary Retinoblastoma Survivors
An observational study of Retinoblastoma, run by Amsterdam UMC, location VUmc at 1 site in Netherlands.
Trial summary
Rationale: Hereditary retinoblastoma survivors have an increased risk to develop second primary tumors (SPT) at a later age (with the highest risk in their teens), especially when they have been irradiated for retinoblastoma. Read more about this study
- Who is it for?
- Ages 8 to 18 years
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- Magnetic resonance imaging
- Type of treatment
- Medical device
- How many people
- 400 participants
- Where
- 1 siteNetherlands
- Study dates
- Oct 2014to Oct 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
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 8 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The potential study population will be irradiated hereditary retinoblastoma survivors in the Netherlands and joining centers from other countries. Currently, there is no screening program for these high-risk patients. The age of patients at highest risk of developing craniofacial SPTs is 8-18 years.
Who may be able to join
- Patients with a history of hereditary retinoblastoma
- Patients that have been treated with external beam radiotherapy for retinoblastoma
- Age of patients at the first MR scan: 8 years or older (≥8 years old), but only as soon as the MR scan can be performed without sedation or general anesthesia, and until and including 18 years of age (≤18 years old).
Who cannot join
- MRI related exclusion criteria:
- Claustrophobia
- Foreign non MR compatible metal objects in the body
- Foreign incompatible metal objects in or close to the head
- Exclusion criterium for additional contrast sequences: Allergic reaction to contrast administration in the past
Trial locations and contacts
1 site in 1 country.
Netherlands1 site, 1 recruiting
- Recruiting
VU University Medical Center
Amsterdam, North Holland, 1081 HV
Marcus C de Jong, MC, MSc, +31204440814, mc.dejong@vumc.nl
About this study
Official title: Multicenter Implementation of MR Imaging Studies in Irradiated Hereditary Retinoblastoma Survivors for Assessment of the Value of Craniofacial MRI Screening for Early Detection of Second Craniofacial Primary Tumors and to Enhance Survival
Rationale: Hereditary retinoblastoma survivors have an increased risk to develop second primary tumors (SPT) at a later age (with the highest risk in their teens), especially when they have been irradiated for retinoblastoma. The investigators hypothesize that regular screening with magnetic resonance imaging (MRI) could lead to early detection of SPTs leading to improved survival.
Objective: To evaluate the potential benefit of craniofacial MRI screening for early detection subclinical secondary cancers in patients previously irradiated for hereditary retinoblastoma.
Read more
Study design: Prospective multicenter non-invasive screening study. The total study duration will be four years of screening plus five years of follow-up.
Study population: Irradiated hereditary retinoblastoma patients 8-18 years old Main study parameters/endpoints: To evaluate the ability of craniofacial MRI for early detection of SPTs, the investigators will determine the sensitivity and specificity of MRI at detecting SPTs in irradiated hereditary retinoblastoma patients.
Nature and extent of the burden and risks associated with participation, benefit and group relatedness: Included patients will undergo yearly craniofacial MRI for a period of 4 years. They will also be asked to fill out a psychological burden assessment form each visit. A potential risk of screening might be associated anticipatory anxiety, but screening also could be reassuring for patients and their parents; the investigators are not sure which will outweigh. False-positive results from MRI screening could lead to unnecessary further diagnostics leading to possible added anxiety and diagnostics (e.g., biopsies). However, this group of patients have a high risk of developing SPTs, with poor 5-year survival statistics. Early detection and therefore treatment of earlier stage (smaller) tumors, might therefore increase survival of this patient group.
- Conditions
- Retinoblastoma
- Being tested
- Magnetic resonance imaging
- Keywords
- Retinoblastoma, Second primary tumor, Screening, Magnetic resonance imaging
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Magnetic resonance imagingDevice
MR imaging protocol should include a thin-slice (3-4 mm) short TI inversion recovery (STIR) sequence or fat-saturated T2-weighted images in axial direction. Furthermore, a 3D-T1 weighted sequence with isotropic voxels will be obtained, with reconstruction in 3 directions. If suspicious lesions are detected, additional MR imaging has to be performed, including contrast-enhanced images.
What is being measured
The primary outcome is the main question the study is designed to answer.
Diagnostic accuracy of MRI for the detection of second primary tumors in hereditary irradiated retinoblastoma
The primary objective of the study is to evaluate the benefit of early detection of craniofacial second primary tumors with MRI in previously irradiated hereditary retinoblastoma survivors. The main outcome measure for assessing the benefit of screening will be the diagnostic accuracy of MRI for detecting craniofacial SPTs: sensitivity (true-positive and false-negative results) and specificity (true-negative and false-positive results).
Measured: 4 years
Secondary outcomes (4)
5-year survival
A secondary objective is to assess the 5-year survival of second primary tumors (SPT) found by screening, SPTs missed by screening, and the non-SPT patients
Measured: 9 years
Descriptive statistics
Recording and monitoring location and characteristics (clinical, radiological, histological, treatment) of patients with SPTs: total number of patients included, median follow-up time + range, number of asymptomatic SPTs detected by screening, number of symptomatic SPTs missed by screening, number of SPTs developed after the end of screening, number of SPTs that were totally resected, location of tumor, type of tumor, size of tumor
Measured: 4 years
Feasability assessment
Assessment of the feasibility of such this screening program: (a) percentage of patients (or family) willing to participate; (b) compliance of patients and their families to the screening program
Measured: 4 years
Assessing the psychosocial burden or benefit
Once a year during the visits, the patient will be asked to fill out the Hospital Anxiety and Depression Scale (HADS).
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2014 (estimated)
- Study startOct 2014
- Primary completionOct 2018 (estimated)
- Study completionOct 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- More trials run by Amsterdam UMC, location VUmcThe sponsor's other registered studies.
- 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
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Amsterdam UMC, location VUmc
- Sponsor type
- University, hospital or other
- Collaborators
- ODAS
- Responsible party
- Principal investigator, Marcus de Jong, Amsterdam UMC, location VUmc
- Investigators
- Jonas A Castelijns, PhD, Study chair, Amsterdam UMC, location VUmc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02329002
- Sponsor's protocol ID
- Rb-SPT-SCR
- Status last verified
- Dec 2014
- First submitted
- 18 Dec 2014
- Last updated
- 31 Dec 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Implementation of MR Imaging Studies in Irradiated Hereditary Retinoblastoma Survivors for Assessment of the Value of Craniofacial MRI Screening for Early Detection of Second Craniofacial Primary Tumors and to Enhance Survival
- ClinicalTrials.gov ID
- NCT02329002
- Acronym / sponsor ID
- Rb-SPT-SCR
- Conditions
- Retinoblastoma
- Keywords
- Retinoblastoma, Second primary tumor, Screening, Magnetic resonance imaging
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- Amsterdam UMC, location VUmcUniversity, hospital or other
- Collaborators
- ODAS
- Study start
- Oct 2014
- Primary completion
- Oct 2018 (estimated)
- Study completion
- Oct 2023 (estimated)
- First posted
- 31 Dec 2014 (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.
Rationale: Hereditary retinoblastoma survivors have an increased risk to develop second primary tumors (SPT) at a later age (with the highest risk in their teens), especially when they have been irradiated for retinoblastoma. The investigators hypothesize that regular screening with magnetic resonance imaging (MRI) could lead to early detection of SPTs leading to improved survival.
Objective: To evaluate the potential benefit of craniofacial MRI screening for early detection subclinical secondary cancers in patients previously irradiated for hereditary retinoblastoma.
Study design: Prospective multicenter non-invasive screening study. The total study duration will be four years of screening plus five years of follow-up.
Study population: Irradiated hereditary retinoblastoma patients 8-18 years old Main study parameters/endpoints: To evaluate the ability of craniofacial MRI for early detection of SPTs, the investigators will determine the sensitivity and specificity of MRI at detecting SPTs in irradiated hereditary retinoblastoma patients.
Nature and extent of the burden and risks associated with participation, benefit and group relatedness: Included patients will undergo yearly craniofacial MRI for a period of 4 years. They will also be asked to fill out a psychological burden assessment form each visit. A potential risk of screening might be associated anticipatory anxiety, but screening also could be reassuring for patients and their parents; the investigators are not sure which will outweigh. False-positive results from MRI screening could lead to unnecessary further diagnostics leading to possible added anxiety and diagnostics (e.g., biopsies). However, this group of patients have a high risk of developing SPTs, with poor 5-year survival statistics. Early detection and therefore treatment of earlier stage (smaller) tumors, might therefore increase survival of this patient group.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 8 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The potential study population will be irradiated hereditary retinoblastoma survivors in the Netherlands and joining centers from other countries. Currently, there is no screening program for these high-risk patients. The age of patients at highest risk of developing craniofacial SPTs is 8-18 years.
Key inclusion criteria (3 listed)
- Patients with a history of hereditary retinoblastoma
- Patients that have been treated with external beam radiotherapy for retinoblastoma
- Age of patients at the first MR scan: 8 years or older (≥8 years old), but only as soon as the MR scan can be performed without sedation or general anesthesia, and until and including 18 years of age (≤18 years old).
Key exclusion criteria (5 listed)
- MRI related exclusion criteria:
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 |
|---|---|---|
| Magnetic resonance imagingMR imaging protocol should include a thin-slice (3-4 mm) short TI inversion recovery (STIR) sequence or fat-saturated T2-weighted images in axial direction. Furthermore, a 3D-T1 weighted sequence with isotropic voxels will be obtained, with reconstruction in 3 directions. If suspicious lesions are detected, additional MR imaging has to be performed, including contrast-enhanced images. | Device | – |
Primary outcome
Diagnostic accuracy of MRI for the detection of second primary tumors in hereditary irradiated retinoblastoma
Time frame: 4 years
The primary objective of the study is to evaluate the benefit of early detection of craniofacial second primary tumors with MRI in previously irradiated hereditary retinoblastoma survivors. The main outcome measure for assessing the benefit of screening will be the diagnostic accuracy of MRI for detecting craniofacial SPTs: sensitivity (true-positive and false-negative results) and specificity (true-negative and false-positive results).
4 secondary outcomes
5-year survival
Time frame: 9 years
Descriptive statistics
Time frame: 4 years
Feasability assessment
Time frame: 4 years
Assessing the psychosocial burden or benefit
Time frame: 4 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2014.
Related trials for Retinoblastoma
RecruitingPhase 1/2
Testing the Addition of an Anti-cancer Drug, Abemaciclib, to the Usual Chemotherapy Treatment (Gemcitabine) for Soft Tissue Sarcoma
Advanced Dedifferentiated Liposarcoma, Advanced Leiomyosarcoma
RecruitingObservational study
Comprehensive Omics Analysis of Pediatric and Adult Solid Tumors and Establishment of a Repository for Related Biological Studies
Sarcoma, Endocrine Tumors
Active, not recruitingPhase 1
EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
Pediatric Solid Tumor, Germ Cell Tumor
Enrolling by invitationObservational study
Effect of Treatment Modality on Psychosocial Functioning of Survivors of Unilateral Retinoblastoma
Survivors of Unilateral Retinoblastoma
Active, not recruitingPhase 1
Safety/Efficacy Study of CID-078 in Patients With Advanced Solid Tumor Malignancies
Advanced Solid Tumor, Metastatic Solid Tumor
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. This study is not recruiting at the moment.
Jonas A Castelijns, PhD study chair
Amsterdam UMC, location VUmc
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 Retinoblastoma, run by Amsterdam UMC, location VUmc at 1 site in Netherlands.
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 3 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 31 Dec 2014. 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 Netherlands. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Marcus C de Jong, MD, MSc (+31204440814, mc.dejong@vumc.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Diagnostic accuracy of MRI for the detection of second primary tumors in hereditary irradiated retinoblastoma (time frame: 4 years). 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 31 Dec 2014.