Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibr… (NCT05677594) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1
United States80 participantsStarted 2021-07-01
Plain-language summary
The goal of this prospective observational study is to learn about the utility of imaging and clinical features in patients with Neurofibromatosis type 1 categorized as high risk for the development of malignant peripheral nerve sheath tumors.
The main objectives are:
* To evaluate the prevalence, multi-parametric imaging features of distinct nodular lesions ("DNLs") and natural history in people with NF1 with clinical and genetic features deemed "high-risk" for malignancy.
* To assess the relationship between individual clinical, genetic and imaging factors that have been suggested to be risk factors for malignant peripheral nerve sheath tumors (MPNST) and the confirmation of atypical neurofibromas (aNF)/ atypical neurofibromatous neoplasm of unknown biologic potential (ANNUBP) or MPNST on pathology.
In this research study, the participants will be asked to undergo whole body MRI, provide blood sample and clinical evaluation annually.
Who can participate
Age range
6 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
Inclusion criteria are person (age \> 6 years) with clinical diagnosis of NF1, ability to undergo whole body MRI without intravenous contrast material considered "high-risk" for MPNST.
The term "high risk" is defined as:
* high pNF burden defined as 1 pNF \> 3 cm or \> 1 pNF
* diagnosis of whole-gene deletion ("microdeletion") of the NF1 gene on genetic testing
* prior history of atypical or malignant PNST
* family or personal history of MPNST or atypical PNST
* prior radiation treatment
Exclusion Criteria:
* Pregnancy
* inability to tolerate MRI or 18F-FDG-PET/CT imaging without anesthesia
* ongoing NF1-related intervention (including systemic steroids) or therapy that may alter the anatomic, metabolic or functional MRI appearance of the PNSTs.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Prevalence of distinct nodular lesions in people with NF1 associated pNF