Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurof… (NCT02544022) | Clinical Trial Compass
RecruitingNot Applicable
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
United States476 participantsStarted 2015-11-13
Plain-language summary
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.
Who can participate
Age range
5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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;
. measures \>=3 cm on longest diameter by visual exam, palpation or 2D MR imaging OR \>=3 mL by volumetric MR imaging.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this trial is focused on developing and validating questionnaires about how NF1 and plexiform neurofibromas affect my daily life — rather than testing a treatment — what would my actual involvement look like, and how much time would it take?
2Because this is a Patient Reported Outcome study, not a treatment trial, would participating in it affect my access to or timing of any active treatment options I might be considering?
3This study is measuring things like reliability and feasibility of these questionnaires, which means it's still in early development — can you help me understand how contributing my experience here might benefit future NF1 patients, even if it doesn't directly help my own care right now?
4Are there any specific symptoms or functional challenges related to my plexiform neurofibromas that this type of study is designed to capture, and do you think my experience would be a good fit for what researchers are trying to learn?
5Since the trial is still recruiting, is this something I could participate in alongside other treatments or monitoring I'm already doing for my NF1?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.