Clinical Trial: NBIAready: Online Collection of Natural History Patient-reported Outcome Measures

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

Official Title: NBIAready: Online Collection of Natural History Patient-reported Outcome Measures

Brief Summary: The purpose of this study is to learn more about a subtype of Neurodegeneration with Brain Iron Accumulation (NBIA) called Pantothenate Kinase-Associated Neurodegeneration (PKAN) by (1) collecting information about how symptoms and findings in NBIA change over time and (2) identifying measures of NBIA that can be used in future clinical trials. Participants will logon to a secure website from home every 6 months for a period of 5 years to electronically complete a set of rating scales as related to their NBIA.

Detailed Summary:
Sponsor: Susan J. Hayflick

Current Primary Outcome: Document the natural history of NBIA and identify new markers of disease progression. [ Time Frame: 5 years ]

The unit of analyses will be the data collected on NBIA milestones and patient-reported outcome measures. Descriptive statistics will be used to characterize the natural history of NBIA. If data support that certain disease milestones occur in a consistent order, or at consistent ages, then these data points will be candidate markers of disease progression. Descriptive statistics will be used to characterize the natural history of NBIA and identify areas in which individuals with NBIA differ from the general population.


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Oregon Health and Science University

Dates:
Date Received: October 23, 2015
Date Started: April 2015
Date Completion: April 2025
Last Updated: September 27, 2016
Last Verified: September 2016