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

Brief Title

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

Official Title

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

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.
    



Study Type

Observational


Primary Outcome

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


Condition

Neurodegeneration With Brain Iron Accumulation (NBIA)


Study Arms / Comparison Groups

 PKAN
Description:  This group consists of individuals diagnosed with PKAN using a combination of MRI, other clinical findings, and PANK2 gene sequencing.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

300

Start Date

April 2015

Completion Date

April 2025

Primary Completion Date

April 2025

Eligibility Criteria

        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.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Susan J Hayflick, MD, 503 494 6838, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02587858

Organization ID

10832


Responsible Party

Sponsor-Investigator

Study Sponsor

Susan J. Hayflick

Collaborators

 Oregon Health and Science University

Study Sponsor

Susan J Hayflick, MD, Principal Investigator, Oregon Health and Science University


Verification Date

October 2020