The NBIA Research Group at OHSU has developed a new study called BPANready. The purpose of this study is to help us better understand the natural history of BPAN, meaning how symptoms appear and change over time. By studying individuals with BPAN, we also hope to identify disease markers that can be used in future clinical trials. A disease marker is any symptom or measurement that happens reliably in a disease, changes predictably with disease progression, and becomes “better” with successful treatment. A disease marker could be an MRI finding, a protein level in the blood, or a rating scale to measure symptoms or function. Natural history studies provide data that serve as the foundation for future drug trials.
Thank you for your interest in BPANready!
1.) Fluency in the English language. The study participant and any guardian or support person who is helping them should be fluent in English. We need to confirm certain milestones using medical records so the study participant’s medical records also need to be in English. Finally, all participants will need to have their doctor complete a clinical assessment tool every 6 months that is written in English.
2.) A computer with Internet access
3.) An email account (please make sure you select an email that you check regularly and will not change for the next few years because we will be sending links to the surveys and reminders through this email address)
4.) A phone number with a reliable connection that you likely won’t be changing soon. If you do change phone numbers, don’t worry, we will ask if your number has changed before you start any follow-up surveys.
5.) A parent or guardian to help you if you are age 0-17 years (it is ok to have someone help you if you are 18 or older, too).
Each study participant will need a “proxy” (parent/guardian) to read the study scales themselves and provide their opinions about the individual with BPAN. The proxy should help care for the participant and know him/her well. The proxy should be the same person for each study visit for the entire duration of the study, since the proxy will be entering his/her opinions about the research participant.
Steps of the BPANready Study
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