Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT06880991
Status
Recruiting
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Conditions

  • Neurofibroma
  • Neurofibromatosis Type 1

Eligibility Criteria

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

Interventions

  • Arm 1 — OTHER
    Participant completion of questionnaires and participation in focus group(s) and/or interview sessions.

Study Details

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.

Key Dates

Start date
Apr 29, 2025
Status verified
Apr 2026
Primary completion
Mar 31, 2027
Completion
Mar 31, 2028

Study Design

Enrollment
110 participants (estimated)

Arms

  • Arm: Cohort 1, Participants with pNF
    Individuals age 8+ years with plexiform neurofibroma (pNF) tumor
  • Arm: Cohort 2, Caregiver of children with pNF
    Caregivers of children (ages 5-17 years) with a plexiform neurofibroma (pNF) tumor
  • Arm: Cohort 3, Participants with cNF
    Individuals age 12+ years with cutaneous neurofibroma (cNF) tumors
  • Arm: Cohort 4, Caregiver of children with cNF
    Caregivers of children (ages 12-17 years) with cutaneous neurofibroma (cNF) tumors

Primary Outcome Measure

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 [ Time Frame: throughout the study ]

Central Contacts

Locations (1)

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

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

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