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

Launched by NATIONAL CANCER INSTITUTE (NCI) · Sep 5, 2015

Keywords

ClinConnect Summary

This clinical trial is focused on improving questionnaires that help understand how neurofibromatosis 1 (NF1) and plexiform neurofibromas (pNFs) affect patients' daily lives, especially regarding pain. The goal is to develop better ways to measure pain, daily activities, and physical abilities for people with these conditions. If you or a family member is 5 years or older and has NF1 with at least one plexiform neurofibroma, you might be eligible to participate in this study.

Participants can expect to be involved in discussions and interviews about their experiences with pain and daily living. The study will happen in two phases. In Phase 1, participants will discuss existing questionnaires in group settings, while younger children will have one-on-one meetings. After improvements are made to the questionnaires, participants may be asked to fill them out to see how well they work. In Phase 2, participants will complete the new questionnaires, either on paper or electronically, and some may fill them out twice to help researchers understand any changes over time. It's important to know that those who have recently started new pain treatments or are involved in other clinical trials may not be eligible to join this study.

Gender

ALL

Eligibility criteria

• * 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.