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

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT06222203
Status
Recruiting
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Conditions

  • Nerve Sheath Neoplasms
  • Neurofibromatosis 1

Eligibility Criteria

Sex
ALL
Age
3 Years - 120 Years
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Oct 9, 2024
Status verified
Jun 2026
Primary completion
Dec 31, 2034
Completion
Dec 31, 2035

Study Design

Enrollment
225 participants (estimated)

Arms

  • Arm: 1 - High-Risk
    Participants with clinical or genetic diagnosis of NF1 AND at least one of the eligibility-required high-risk characteristics
  • Arm: 2 - Low-Risk
    Participants with clinical or genetic diagnosis of NF1 AND none of the eligibility-required high-risk characteristic
  • Arm: 3 - Caregiver
    Parents or guardians of participants 8-17 years old in High-Risk or Low-Risk Cohorts

Primary Outcome Measure

Assess feasibility of the study algorithm in identifying atypical neurofibromas (ANs), atypical neurofibromatous neoplasms of unknown biologic potential (ANNUBPs), CDKN2A/B mutated lesions, and/or malignant peripheral nervous sheath tumors (MPNS... [ Time Frame: Throughout the study ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
National Cancer Institute Referral Office
888-624-1937

Find similar trials in Bethesda, MD

By research site
National Institutes of Health Clinical Center· Bethesda, MD

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