Clinical Trial: Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (PNs)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (PNs)

Brief Summary:

Background:

People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (PNs) 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 PNs.

Eligibility:

People ages 5 and older with NF1 and a PN

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 PNs affect their life. They will have group discussions of up to 8 people of a similar age with NF1 and PNs, 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 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

Detailed Summary:

Background:

  • Neurofibromatosis 1 (NF1) is a genetic disease with multiple clinical manifestations, including plexiform neurofibromas (PN) that can cause pain and may significantly impact daily functioning and quality of life (QOL).
  • Patient-reported outcomes (PROs) are useful in trials for conditions that are disabling and chronic like NF1, where symptom reduction and improved functioning and QOL currently are important treatment outcomes, which may occur with PN shrinkage.
  • A critical step toward approval of drugs to treat PNs is to evaluate clinical benefit in conjunction with a reduction in tumor volume as assessed by imaging endpoints.
  • The FDA requests the use of PROs in NF1 clinical atrials, especially for assessing changes in symptoms, such as pain.
  • Currently, no valid PRO measures exist that are specific to the NF1 population to assess PN pain, its functional impact on an individual s life, or overall physical functioning, which may be affected by PNs.

Objectives:

  • Phase 1: Qualitative Evaluation

    --Primary Objective:

    • 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 q
      Sponsor: National Cancer Institute (NCI)

      Current Primary Outcome: Evaluate final versions of the NRS-11, PII, and PROMIS-PF measures on reliability, validity, sensitivity to change, and feasibility in individualswith NF1 and PNs [ Time Frame: 8 months ]

      Original Primary Outcome: Same as current

      Current Secondary Outcome:

      • To provide normative data on these measures of pain intensity, pain interference, and physical functioning in individuals with NF1 and PNs [ Time Frame: 8 months ]
      • To assess the psychometric properties and provide normative data for the Adult version of the Impact of Pediatric Illness (IPI) Scale [ Time Frame: 8 months ]


      Original Secondary Outcome:

      • To provide normative data on these measures of pain intensity, pain interference, and physical functioning in individuals with NF1 and PNs, [ Time Frame: 8 months ]
      • To assess the psychometric properties and provide normative data for the Adult version of the Impact of Pediatric Illness (IPI) Scale [ Time Frame: 8 months ]


      Information By: National Institutes of Health Clinical Center (CC)

      Dates:
      Date Received: September 5, 2015
      Date Started: September 3, 2015
      Date Completion: December 31, 2018
      Last Updated: April 21, 2017
      Last Verified: September 26, 2016