RecruitingNCT06222203

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)


Sponsor

National Cancer Institute (NCI)

Enrollment

225 participants

Start Date

Oct 9, 2024

Study Type

OBSERVATIONAL

Conditions

Summary

Background: Neurofibromatosis type 1 (NF1) is a genetic disease that can cause many symptoms. About half of people with NF1 will develop benign (noncancerous) tumors along nerves in the skin, brain, and other parts of the body. Sometimes, though, these tumors can become cancerous. Researchers do not yet know how to predict which tumors will become cancerous. Objective: To test a new method for predicting which benign NF1 tumors will become cancerous. Eligibility: People aged 3 years and older with a clinical or genetic diagnosis of NF1. Design: * Participants will be screened with a review of their medical history. All participants will have a baseline visit. They will have bood tests and imaging scans. They will have a physical exam. They will answer questions about their family history. Participants aged 8 years and older will take tests of their thinking skills and their emotional health. * Some participants may be asked to undergo more tests. These may include another type of imaging scan and a biopsy: A small sample of tissue may be removed from the tumor. * Participants will be divided into two groups: those believed to be at low risk and those believed to be at high risk of developing cancer. * Participants in the high-risk group will be asked to return for their next visit in 1 month to 3 years. * Participants in the low-risk group will be asked to return for their next visit in 6 months to 5 years. * Participants may also have follow-up visits by phone throughout the study. They will be in the study for 10 years.


Eligibility

Min Age: 3 YearsMax Age: 120 Years

Plain Language Summary

Simplified for easier understanding

This study is monitoring people with Neurofibromatosis Type 1 (NF1) — a genetic condition that causes tumors to grow on nerves — for early signs of nerve tumors turning cancerous (malignant). Regular MRI scans will be used to track any changes in existing tumors. **You may be eligible if...** - You are 3 years old or older - You have a clinical or genetic diagnosis of NF1 (including mosaic or segmental forms) - You may or may not have specific higher-risk features, such as a family history of cancerous nerve tumors or a large nerve tumor burden - A parent or legal guardian can provide consent if you are a minor **You may NOT be eligible if...** - You are unable or unwilling to undergo MRI scans Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(1)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

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NCT06222203


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