Recruiting

Observational Study

Sponsor:

National Cancer Institute (NCI)

Code:

NCT06880991

Conditions

Neurofibromatosis Type 1

Neurofibroma

Eligibility Criteria

Sex: All

Age: 5 - 70+

Healthy Volunteers: Not accepted

Interventions

Arm 1

Study Details

Brief 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.

Conditions

Neurofibromatosis Type 1

Neurofibroma

Study ID

NCT06880991

Start date

Apr 29, 2025

Status verified date

Aug 12, 2026

Completion date

Mar 31, 2028

Anticipated

Primary completion date

Mar 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 5 - 70+

Healthy Volunteers: Not accepted

  • 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 ensure that the measures are appropriate for people from a variety of backgrounds.

\* Underrepresented groups are defined as people who identify as African American or Black, American Indian, Alaska Native, Hispanic/Latine, Native Hawaiian, and other Pacific Islander.
  • If saturation has been met within a particular age group, potential participants from that completed age group will no longer be eligible.

Study Design

Enrollment

110 participants

Anticipated

Interventions and Outcome Measures

Arms

Cohort 1, Participants with pNF

Individuals age 8+ years with plexiform neurofibroma (pNF) tumor

Cohort 2, Caregiver of children with pNF

Caregivers of children (ages 5-17 years) with a plexiform neurofibroma (pNF) tumor

Cohort 3, Participants with cNF

Individuals age 12+ years with cutaneous neurofibroma (cNF) tumors

Cohort 4, Caregiver of children with cNF

Caregivers of children (ages 12-17 years) with cutaneous neurofibroma (cNF) tumors

Interventions

Arm 1

Participant completion of questionnaires and participation in focus group(s) and/or interview sessions.

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 and Locations

Central contacts

Staci M Peron, Ph.D.

(240) 760-6025martins@mail.nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

National Cancer Institute Referral Office

888-624-1937ncimo_referrals@mail.nih.gov

More Information

Sponsor

National Cancer Institute (NCI)

Last update posted

Aug 14, 2026

Last verified

Aug 12, 2026

Keywords

  • Qualitative
  • Focus Groups
  • Instrument Development

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by National Cancer Institute (NCI) on 2026-08-14.