Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concer… (NCT06880991) | Clinical Trial Compass
RecruitingNot Applicable
Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study
United States110 participantsStarted 2025-04-29
Plain-language summary
Background:
Neurofibromatosis 1 (NF1) is a disease that causes tumors to grow along the nerves. These include plexiform neurofibromas (pNF) and cutaneous neurofibromas (cNF). Both pNF and cNF can be visible to other people. These tumors can affect a person s appearance and quality of life. Researchers want to be able to assess changes in appearance before and after treatment for NF1 tumors.
Objective:
To see if two questionnaires can help assess people's ratings about the appearance of their pNF and cNF tumors.
Eligibility:
People aged 8 years and older with pNF and people 12 years and older either with cNF or both pNF and cNF. Adult caregivers of children with pNF and cNF are also needed.
Design:
Participants will complete questionnaires on paper or by phone, computer, or tablet. They will answer questions about how they look, how they feel, and how they feel about the way they look.
Participants will meet in at least 1 remote focus group or individual interview. The meeting will last about 1 hour. Each group will include 3 to 5 people, organized by age: 8 to 11 years, 12 to 17 years, 18 to 29 years, and over 30 years. Adult caregivers will meet in a group with other caregivers.
They will discuss their NF1 symptoms; how their tumors look; how they feel about the way their tumors look; and their daily activities. They will give their opinions about 2 questionnaires about appearance.
The group and individual meetings will be audio-recorded and transcribed. Information that can reveal individual identities will be removed.
Who can participate
Age range
5 Years – 120 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:
* Participants must self-report a diagnosis of NF1 OR be the caregiver of a child with NF1 (5-17 years old with plexiform neurofibroma \[pNF\] or 12-17 years old with cutaneous neurofibroma \[cNF\])
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)
* Access to a 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
* Since we will aim to have no less than 25 percent of participants from underrepresented\* groups, individuals from represented groups may not be able to participate after we have reached the maximum target for represented groups. Consistent with best practices for scale development and validation, this will help ensur…
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
Evaluate new tumor visibility (DRS-pNF, DRS-cNF self-report/parent-report); to select the most appropriate items to assess these constructs in NF1 based on qualitative feedback from children and adults with NF1 and visible pNF or cNF