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

Neurodegeneration With Brain Iron Accumulation (NBIA) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02587858
• Other study ID #: 10832
• Status: Recruiting
• Start date: April 2015
• Est. completion date: April 2025

Study information

• Verified date: October 2020
• Source: Oregon Health and Science University
• Contact: Alison C Freed, BA
• Phone: 503 494 6838
• Email: freeal[@]ohsu.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 300
• Est. completion date: April 2025
• Est. primary completion date: April 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Diagnosis of PKAN, PLAN or BPAN confirmed by gene testing and/or clinical features.

• Ability to access a computer with internet services or a phone approximately once every 6 months for up to 10 years to enter data.

Exclusion Criteria:

• Individuals who are not fluent in reading and communicating in English.

Related Conditions & MeSH terms

• Nerve Degeneration
• Neurodegeneration With Brain Iron Accumulation (NBIA)
• Pantothenate Kinase-Associated Neurodegeneration

Locations

Country	Name	City	State
United States	Oregon Health & Science University	Portland	Oregon

Sponsors (2)

Lead Sponsor	Collaborator
Susan J. Hayflick	Oregon Health and Science University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Document the natural history of NBIA and identify new markers of disease progression.	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.	5-10 years

External Links

•   Official website of the OHSU NBIA research group