Actively Recruiting
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
Led by National Cancer Institute (NCI) · Updated on 2026-06-01
476
Participants Needed
6
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying people with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (pNFs), a condition that can cause pain and affect daily activities. The study aims to improve questionnaires that measure pain, daily living, and physical functioning for individuals with NF1 and pNFs. This research involves two phases, focusing on developing patient-reported outcome measures that can be used in clinical trials to evaluate treatment benefits. The study includes two phases: Phase 1 involved group discussions or one-on-one interviews with children aged 5 to 7 and their parents to review and improve existing pain assessment questionnaires. Phase 2, now complete, involved participants completing the new or revised questionnaires, either on paper or electronically. Some participants completed the forms twice, both in clinic and at home, and those starting or increasing pain treatment completed forms before and after treatment changes. Participants will be involved in completing questionnaires about their pain and daily functioning. These include qualitative group discussions and individual interviews in Phase 1, and repeated questionnaire completion in Phase 2 to assess reliability and validity. The study measures include reliability and feasibility of questionnaires over about eight months. Participants may also provide input on the electronic format of the questionnaires. The total study duration varies by participant depending on phase involvement.
CONDITIONS
Brief Title
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented neurofibromatosis 1 (NF1) by NIH clinical criteria or molecular testing
- At least one plexiform neurofibroma (pNF) measuring 3 cm or larger by exam or imaging, or 3 mL or more by volumetric MRI
- Age 5 years or older for Phase 1; age 8 years or older for Phase 2
- Ability to understand, read, and speak English
- Ability and willingness to sign informed consent (patient or parent/guardian)
- For Phase 1 focus groups: recent pNF-related pain with minimum pain level requirements or use of prescription pain medication
- For Phase 2: patients with or without recent pNF-related pain as specified
- Primary caregivers (parents, guardians, grandparents) aged 18 or older of participants 17 years or younger
You will not qualify if you...
- Severe cognitive or behavioral impairments preventing cooperation with study procedures
- Enrollment in a MEK inhibitor trial within the past 12 months
- Started a new pain treatment regimen (medication, psychosocial therapy, physical therapy, etc.) within 3 months prior to enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 2 weeks
Participants complete electronic versions of pain intensity and pain interference questionnaires over two weeks to assess reliability, validity, and collect normative data.
2 visits (in-person or electronic completion) over two consecutive weeks
Trial Site Locations
Total: 6 locations
1
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Completed
2
University of Chicago
Chicago, Illinois, United States, 60637
Active, Not Recruiting
3
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Active, Not Recruiting
4
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
5
Cincinnati Children's Hospital and Medical Center Institution
Cincinnati, Ohio, United States, 45229-3039
Completed
6
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
P
Pamela L Wolters, Ph.D.
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
5
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