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

Status: Recruiting
Location: See location...
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 120
Healthy Volunteers: f
View:

⁃ Parent Cohort

• Parent or guardian of pediatric individuals (8-17 years old) in High-Risk or Low-Risk Cohorts.

• The ability of the parent/guardian or LAR to understand and the willingness to sign a written consent document for parent/guardian participation in this study.

Locations
United States
Maryland
National Institutes of Health Clinical Center
RECRUITING
Bethesda
Contact Information
Primary
Jennifer E Derise
jennifer.derise@nih.gov
(240) 575-8520
Backup
Brigitte C Widemann, M.D.
widemanb@mail.nih.gov
(240) 760-6203
Time Frame
Start Date: 2024-10-09
Estimated Completion Date: 2035-12-31
Participants
Target number of participants: 225
Treatments
1 - High-Risk
Participants with clinical or genetic diagnosis of NF1 AND at least one of the eligibility-required high-risk characteristics
2 - Low-Risk
Participants with clinical or genetic diagnosis of NF1 AND none of the eligibility-required high-risk characteristic
3 - Caregiver
Parents or guardians of participants 8-17 years old in High-Risk or Low-Risk Cohorts
Sponsors
Leads: National Cancer Institute (NCI)

This content was sourced from clinicaltrials.gov