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 2026-04-24
476
Participants Needed
6
Research Sites
580 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (pNFs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning. Objectives: To examine and improve questionnaires about daily living for people with NF1 and pNFs. Eligibility: People ages 5 and older with NF1 and a pNF Design: Participants will be screened with medical history. This study will have 2 phases. Phase 1 participants will talk about existing pain assessment questionnaires and how pNFs affect their life. They will have group discussions of up to 8 people of a similar age with NF1 and pNFs, or the parents of children with it. These will last about 90 minutes. Children ages 5 to 7 and their parents will have one-on-one meetings instead. These will last about 45 minutes. Discussions will be audiotaped. After the questionnaires have been changed, individual interviews will discuss the new wording, instructions, questions, and electronic format of the new forms. Phase 2 is now complete. Phase 1 participants may be invited to Phase 2. Phase 2 participants will complete the new questionnaires. These may be pen-and-paper or electronic. The questionnaires will take about 30 minutes for adults and teens. Children will work one-on-one with a staff member and may need up to 45 minutes. A small group of participants will be complete the forms twice-in clinic and 1 month later at home. Also, a small group who start a new pain treatment or have a dose increase in their treatment will complete the forms twice-before the treatment change and 1 month later.
CONDITIONS
Official 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 genetic testing
- At least one plexiform neurofibroma (pNF) measuring 3 cm or more by exam or imaging
- For phase 1, participants aged 5 years or older
- For phase 2, participants aged 8 years or older
- Ability to understand, read, and speak English
- Willingness to sign informed consent
- For phase 1 focus groups, recent pNF-related pain with minimum pain level of 3 on NRS-11 or use of prescription pain medication with pain level of 1 or more
- For phase 2 patients with pain, recent experience of pain in target tumor area
- For phase 2 patients without pain, no recent pain in target tumor area
- Primary caregivers (parents, guardians, grandparents) aged 18 or older of participants 17 years or younger may participate to provide parent reports
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
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.
CONTACT
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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