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Observational Study on Patient-Reported Outcomes for NF1 and Plexiform Neurofibromas

This study is for people with Neurofibromatosis 1 (NF1) and plexiform neurofibromas (pNFs). It aims to improve questionnaires that measure how pain and pNFs affect daily life. Currently, there aren't good questionnaires specifically for NF1 patients to assess pain and its impact. Researchers want to make sure these questionnaires accurately capture your experiences. To join, you must have documented NF1 (either by clinical criteria or a genetic mutation in the NF1 gene) and at least one pNF. The study will look at how reliable and practical these improved questionnaires are over 8 months. This study does not involve any new treatments, but focuses on improving how we understand and measure the impact of NF1 and pNFs on your life.

Study design
This is an observational study planning to enroll 476 participants. It has two phases, with the first phase involving group discussions.
What's involved
Participants will be screened with a medical history. Phase 1 involves group discussions of up to 8 people, lasting about 90 minutes.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoints (reliability and feasibility) are measured at 8 months, suggesting follow-up for at least that duration.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT02544022

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)

Recruiting
Not specifiedAges 5+Observational
National Cancer Institute (NCI)
~476 participants
Updated 2026-09-01 on ClinicalTrials.gov

At a glance

Recruiting sites
2 of 6 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Realiability
Measured over 8 months
+1 more outcome measured

Conditions

Neurofibromatosis 1
Plexiform Neurofibromas

Explore related trials

Neurofibromatosis Type 1Plexiform Neurofibroma

Where it's being run

6 sites across 5 states
Maryland2
District of Columbia1
Illinois1
Ohio1
Pennsylvania1

Study leadership

  • Pamela L Wolters, Ph.D. · PRINCIPAL_INVESTIGATOR · National Cancer Institute (NCI)

Who to contact

Pamela L Wolters, Ph.D.
[email protected]
(240) 760-6035
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Documented NF1 either by NIH clinical criteria or molecularly-proven mutation in the
For phase 1, Age \>=5 years. (complete)
For phase 2, Age \>= 8 years
Ability of subject or parent or guardian to understand and the willingness to sign a written informed consent document.
Participants must be able to understand, read, and speak the English language.
For phase 1 focus groups only, patients need to report experiencing pNF related pain recently with a minimum pain level of 3 on the current NRS-11 or report taking prescription medication that reduces pain and experiencing pNF related pain recently with a minimum pain level of 1 on the current NRS-11. (complete)
For phase 2 patients with pain, patients need to report recently experiencing at least a minimal amount of pNF-related pain. Specifically, they will be asked if they recently experienced any pain in a target tumor area and will have to respond yes to be eligible.
For phase 2 patients without pain, patients need to report no recent pNF-related pain. Specifically, they will be asked if they recently experienced any pain in a target tumor area and will have to respond no to be eligible.
Primary caregiver (i.e. parent,guardian, grandparent) who is \>= 18 years old of participating subject \<= 17 years old
Participants must be able to understand, read, and speak the English language

Exclusion

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

What this trial measures

  • Realiability8 months

    To evaluate final versions of the NRS-11, PII, and PROMIS-PF measures on reliability, validity, sensitivity to change, and feasibility in individuals with NF1 and PNs.

  • Feasability8 months

    To evaluate current modifications and the need for any additional modifications to existing measures of pain intensity (Numeric Rating Scale; NRS-11) and pain interference (Pain Interference Index; PII) and select the most appropriate items to measure physical functioning (PROMIS Physical Functioning; PROMIS-PF) in NF1 based on qualitative feedback from patients with NF1, PNs and pain to use as endpoints in clinical trials for individuals with NF1 and PNs. (completed)