NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
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|ClinicalTrials.gov Identifier: NCT02587858|
Recruitment Status : Recruiting
First Posted : October 27, 2015
Last Update Posted : October 8, 2020
The purpose of this study is to learn more about Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders. Data is being collected on three types of NBIA disorders:
Pantothenate Kinase-Associated Neurodegeneration (PKAN), PLA2G6-associated Neurodegeneration (PLAN) and Beta-propeller Protein-associated Neurodegeneration (BPAN). The study will (1) collect information about how symptoms and findings in NBIA change over time and (2) identify measures of NBIA that can be used in future clinical trials. Participants will follow links to a secure website every 6 months for a period of 5-10 years to electronically complete a set of rating scales as related to their NBIA disorder.
|Condition or disease|
|Neurodegeneration With Brain Iron Accumulation (NBIA)|
|Study Type :||Observational|
|Estimated Enrollment :||300 participants|
|Official Title:||NBIAready: Online Collection of Natural History Patient-reported Outcome Measures|
|Study Start Date :||April 2015|
|Estimated Primary Completion Date :||April 2025|
|Estimated Study Completion Date :||April 2025|
This group consists of individuals diagnosed with PKAN using a combination of MRI, other clinical findings, and PANK2 gene sequencing.
This group consists of individuals diagnosed with PLAN using a combination of MRI, other clinical findings and PLA2G6 gene sequencing.
This group consists of individuals diagnosed with BPAN using a combinatino of MRI, other clinical findings, and WDR45 gene sequencing.
- Document the natural history of NBIA and identify new markers of disease progression. [ Time Frame: 5-10 years ]The unit of analyses will be the data collected on NBIA milestones and patient-reported outcome measures. Latent Growth Curve (LGC) modeling 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. LGC will be used to characterize the natural history of NBIA and identify areas in which individuals with NBIA differ from the general population.
Biospecimen Retention: Samples With DNA
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): NCT02587858
|Contact: Alison C Freed, BA||503 494 firstname.lastname@example.org|
|Contact: Allison M Gregory, MS, CGC||503 494 email@example.com|
|United States, Oregon|
|Oregon Health & Science University||Recruiting|
|Portland, Oregon, United States, 97239-3098|
|Contact: Alison C Freed, BA 503-494-6838 firstname.lastname@example.org|
|Contact: Allison M Gregory, MS, CGC 503 494 4344 email@example.com|
|Principal Investigator: Susan J Hayflick, MD|
|Principal Investigator:||Susan J Hayflick, MD||Oregon Health and Science University|