Development of Patient-Reported Outcome Measures Assessing Tumor-Related Disfigurement and Appearance Concerns in Neurofibromatosis Type 1

Launched by NATIONAL CANCER INSTITUTE (NCI) · Mar 17, 2025

Keywords

ClinConnect Summary

This clinical trial is studying how two questionnaires can help understand how people with Neurofibromatosis Type 1 (NF1) feel about the appearance of tumors that grow on their nerves. These tumors, called plexiform neurofibromas (pNF) and cutaneous neurofibromas (cNF), can be visible and may affect a person's self-esteem and daily life. The goal is to gather information from patients and their caregivers to improve the way we assess the impact of these tumors on appearance before and after treatment.

To participate, individuals need to be at least 8 years old if they have pNF or at least 12 years old if they have cNF or both types of tumors. Caregivers of children with NF1 are also welcome to join. Participants will complete questionnaires about their feelings and experiences related to their appearance and participate in discussions in small groups or individual interviews, which will last about an hour. These sessions will allow them to share their thoughts and opinions about their symptoms and the questionnaires being tested. It's important for participants to be comfortable discussing their condition in English and have access to a device with the internet.

Gender

ALL

Eligibility criteria

• * INCLUSION CRITERIA:
• • Participants must self-report a diagnosis of NF1 OR be the caregiver of a child with NF1 (5-17 years old with \[pNF\] or 12-17 years old child with \[cNF\])
• The following for the participant or the caregiver of a child, as appropriate:
• • Participants with NF1 must self-report a pNF and/or cNF tumor(s) that is visible to that individual OR others
• • Caregivers of a child with NF1 must report a pNF and/or cNF tumor(s) that the child has that is visible to the child OR others
• * Age requirements:
• • \>= 8 years old (participants with pNF)
• • \>= 12 years old (participants with cNF)
• • \>= 12 years old (participants with pNF and cNF)
• • \>= 18 years (caregivers only)
• • Access to device with internet
• • Ability to understand English and comfort discussing their medical condition in English
• • The ability of adult participant or caregiver of minor participants to understand and the willingness to sign a written informed consent document
• EXCLUSION CRITERIA:
• • Physical or cognitive limitations that would prevent them from being able to participate in a focus group or interview, or unwillingness to do so
• • If saturation has been met within a particular age group, potential participants from that completed age group will no longer be eligible.