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

Status unconfirmed

Conditions studied: Neurodegeneration With Brain Iron Accumulation (NBIA)

In brief

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.

Key facts

Study ID

NCT02587858

Run by

Susan J. Hayflick

People needed

300

Starts

2015-04-01

Expected to finish

2025-04-01

Last updated by the study team

2020-10-08

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

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

Where it is running

• Oregon Health & Science University — Portland, Oregon, United States (enrolling)

Full record on ClinicalTrials.gov

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