Improving questionnaires for pain and daily life in neurofibromatosis 1 patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study focuses on enhancing patient-reported outcome measures for individuals with neurofibromatosis 1 (NF1) and plexiform neurofibromas (pNFs). It involves two phases: the first phase includes group discussions and interviews to gather qualitative feedback on existing pain assessment tools, while the second phase will evaluate the psychometric properties of the revised questionnaires. The goal is to create valid measures that accurately reflect the pain and functional impact of pNFs on patients' lives, which can be used in future clinical trials. Participants will be ages 5 and older, and the study will take place at multiple prominent medical institutions.

Why it matters

Who can join: the full rules

* SUBJECT INCLUSION CRITERIA:
* Documented NF1 either by NIH clinical criteria or molecularly-proven mutation in the

NF1 gene, PER the Neurofibromatosis Diagnostic Criteria AND \>=1 plexiform neurofibroma in any location that is either symptomatic or asymptomatic, and is defined by the following:

1. a neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches OR a spinal neurofibroma that involves two or more levels with connection between the levels or extending laterally along the nerve OR a skin thickness neurofibroma;
2. measures \>=3 cm on longest diameter by visual exam, palpation or 2D MR imaging OR \>=3 mL by volumetric MR imaging.

   * 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 INCLUSION CRITERIA:

* 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 CRITERIA:

* 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's running

• National Institutes of Health Clinical Center
  Bethesda, Maryland, United States Recruiting
• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting
• Children's National Medical Center
  Washington D.C., District of Columbia, United States Completed
• University of Chicago
  Chicago, Illinois, United States Active not recruiting
• Johns Hopkins University
  Baltimore, Maryland, United States Active not recruiting
• Cincinnati Children's Hospital and Medical Center Institution
  Cincinnati, Ohio, United States Completed

Study contacts

• Principal investigator: Pamela L Wolters, Ph.D. — National Cancer Institute (NCI)
• Study coordinator: Pamela L Wolters, Ph.D.
• Email: woltersp@mail.nih.gov
• Phone: (240) 760-6035

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.