Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (PNs)
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|ClinicalTrials.gov Identifier: NCT02544022|
Recruitment Status : Recruiting
First Posted : September 9, 2015
Last Update Posted : May 13, 2021
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.
To examine and improve questionnaires about daily living for people with NF1 and PNs.
People ages 5 and older with NF1 and a PN
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 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.
|Condition or disease|
|Neurofibromatosis 1 Plexiform Neurofibromas|
|Study Type :||Observational|
|Estimated Enrollment :||486 participants|
|Official Title:||Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (PNs)|
|Actual Study Start Date :||November 13, 2015|
|Estimated Primary Completion Date :||February 28, 2023|
|Estimated Study Completion Date :||February 28, 2023|
1/Phase 1 Focus Group
Patients with NF1 who have PNs and report experiencing PN related pain and parents ofthese patients. (completed)
2/Phase 1 Patients
Patients with NF1 who have PNs
3/Phase 1 Parent
Parents of patients in cohort 2
4/Phase 2 Patients
Patients with NF1 who have PNs
5/Phase 2 Parents
Parents of patients enrolled in cohort 4
- Feasability [ Time Frame: 8 months ]To evaluate current modifications and the need for any additional modifications to existing measures of pain intensity (Numeric Rating Scale; NRS-ll) and pain interference (Pain Interference Index; PII) and select the most appropriate items to measure physical functioning (PROMIS Physical Functioning; PROMIS-PF) in NFl based on qualitative feedback from patients with NFl, PNs and pain to use as endpoints in clinical trials for individuals with NFl and PNs.
- Realibility [ Time Frame: 8 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.
- Convert the measures into electronic format [ Time Frame: 8 months ]To provide normative data on these measures of pain intensity, pain interference, and physical functioning in individuals with NFl and PNs,
- To provide normative data [ 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02544022
|Contact: Pamela L Wolters, Ph.D.||(240) email@example.com|
|United States, District of Columbia|
|Children's National Medical Center||Recruiting|
|Washington, District of Columbia, United States, 20010|
|Contact: Karin Walsh 202-476-3923 firstname.lastname@example.org|
|United States, Illinois|
|University of Chicago||Recruiting|
|Chicago, Illinois, United States, 60637|
|Contact: James Tonsgard 773-702-6487 email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office 888-624-1937|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center||Recruiting|
|Cincinnati, Ohio, United States, 45229-3039|
|Contact: Elizabeth Schorry 513-636-4760 firstname.lastname@example.org|
|Principal Investigator:||Pamela L Wolters, Ph.D.||National Cancer Institute (NCI)|