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

Part of paid clinical trials in Washington D.C., District of Columbia.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT02544022
Status
Recruiting
Apply to this trial

Conditions

  • Neurofibromatosis 1
  • Plexiform Neurofibromas

Eligibility Criteria

Sex
ALL
Age
5 Years - N/A
Healthy Volunteers
Accepted

Study Details

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 Dates

Start date
Nov 13, 2015
Status verified
Nov 2025
Primary completion
Jun 30, 2026
Completion
Dec 31, 2026

Study Design

Enrollment
476 participants (estimated)

Arms

  • Arm: 1/Phase 1 Focus Group
    Patients with NF1 who have PNs and report experiencing pNF related pain and parents of these patients. (completed)
  • Arm: 2/Phase 1 Patients
    Patients with NF1 who have pNFs(completed)
  • Arm: 3/Phase 1 Parent
    Parents of patients in cohort 2 (completed)
  • Arm: 4/Phase 2 Patients
    Patients with NF1 who have pNFs and recent pNF-related pain
  • Arm: 5/Phase 2 Parents
    Parents of patients (ages 8-17 years) enrolled in cohort 4

Primary Outcome Measure

Realiability [ Time Frame: 8 months ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
Children's National Medical CenterWashington D.C.District of Columbia20010-
University of ChicagoChicagoIllinois60637-
Johns Hopkins UniversityBaltimoreMaryland21287-
National Institutes of Health Clinical CenterBethesdaMaryland20892
National Cancer Institute Referral Office
888-624-1937
Cincinnati Children's Hospital and Medical Center InstitutionCincinnatiOhio45229-3039-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Ratnakar Patti
[email protected]

Find similar trials in Washington D.C., DC

By research site
Children's National Medical Center· Washington D.C., DCUniversity of Chicago· Chicago, ILJohns Hopkins University· Baltimore, MDNational Institutes of Health Clinical Center· Bethesda, MDCincinnati Children's Hospital and Medical Center Institution· Cincinnati, OHChildren's Hospital of Philadelphia· Philadelphia, PA

Related Studies