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

Description

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.

Conditions

Neurodegeneration With Brain Iron Accumulation (NBIA)

Study Overview

Study Details

Study overview

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.

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

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

Condition
Neurodegeneration With Brain Iron Accumulation (NBIA)
Intervention / Treatment

-

Contacts and Locations

Portland

Oregon Health & Science University, Portland, Oregon, United States, 97239-3098

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

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

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Susan J. Hayflick,

Susan J Hayflick, MD, PRINCIPAL_INVESTIGATOR, Oregon Health and Science University

Study Record Dates

2025-04