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

Recruiting

• Conditions: Neurodegeneration With Brain Iron Accumulation (NBIA)

• Registration Number: NCT02587858
• Lead Sponsor: Susan J. Hayflick

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-04
• Study First Submitted: 2015-10-23
• Study First Submitted QC: 2015-10-23
• Study First Posted: 2015-10-27
• Status Verified: 2020-10
• Last Update Submitted: 2020-10-05
• Last Update Posted: 2020-10-08
• Primary Completion: 2025-04
• Study Completion: 2025-04

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 300

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Document the natural history of NBIA and identify new markers of disease progression.	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.

Trial Locations

Locations (1)

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States