Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
Recruiting now
Conditions studied: Neurofibromatosis 1, Plexiform Neurofibromas
In brief
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.
Key facts
- Study ID
- NCT02544022
- Run by
- National Cancer Institute (NCI)
- People needed
- 476
- Starts
- 2015-11-13
- Expected to finish
- 2027-12-31
- Last updated by the study team
- 2026-07-17
Who can join
Age: 5 and older. Sex: any. Healthy volunteers: accepted.
You may not qualify if…
- Patients with severe cognitive or behavior impairments who, in the judgment of the investigators, would not be able to cooperate with the study procedures will be excluded.
- Patients cannot be newly enrolled on a clinical trial to treat their pNF or cannot have started a new pain treatment regimen (e.g., medication, psychosocial therapy, physical therapy, etc.) at the time of enrollment. Specifically, patients will be ineligible if they were enrolled on a MEK inhibitor trial in the past 12 months or began a new pain
- medication or treatment within the past 3 months prior to enrollment on this study.
Where it is running
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
- Children's National Medical Center — Washington D.C., District of Columbia, United States
- University of Chicago — Chicago, Illinois, United States
- Johns Hopkins University — Baltimore, Maryland, United States
- Cincinnati Children's Hospital and Medical Center Institution — Cincinnati, Ohio, United States
Full record on ClinicalTrials.gov
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