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

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ClinicalTrials.gov Identifier: NCT02587858
Recruitment Status : Recruiting
First Posted : October 27, 2015
Last Update Posted : October 8, 2020
Sponsor:
Collaborator:
Oregon Health and Science University
Information provided by (Responsible Party):
Susan J. Hayflick, Oregon Health and Science University

Study Description
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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.


Condition or disease
Neurodegeneration With Brain Iron Accumulation (NBIA)

Study Design
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Study Type : Observational
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
Study Start Date : April 2015
Estimated Primary Completion Date : April 2025
Estimated Study Completion Date : April 2025

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort
PKAN
This group consists of individuals diagnosed with PKAN using a combination of MRI, other clinical findings, and PANK2 gene sequencing.
PLAN
This group consists of individuals diagnosed with PLAN using a combination of MRI, other clinical findings and PLA2G6 gene sequencing.
BPAN
This group consists of individuals diagnosed with BPAN using a combinatino of MRI, other clinical findings, and WDR45 gene sequencing.


Outcome Measures
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Primary Outcome Measures :
  1. Document the natural history of NBIA and identify new markers of disease progression. [ Time Frame: 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.


Biospecimen Retention:   Samples With DNA
BPAN participants will be asked for blood and saliva samples.

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The PKAN group consists of international individuals diagnosed with PKAN. The PLAN group consists of international individuals diagnosed with PLAN. The BPAN group consists of international individuals diagnosed with BPAN.
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02587858


Contacts
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Contact: Alison C Freed, BA 503 494 6838 freeal@ohsu.edu
Contact: Allison M Gregory, MS, CGC 503 494 4344 gregorya@ohsu.edu

Locations
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United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239-3098
Contact: Alison C Freed, BA    503-494-6838    freeal@ohsu.edu   
Contact: Allison M Gregory, MS, CGC    503 494 4344    gregorya@ohsu.edu   
Principal Investigator: Susan J Hayflick, MD         
Sponsors and Collaborators
Susan J. Hayflick
Oregon Health and Science University
Investigators
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Principal Investigator: Susan J Hayflick, MD Oregon Health and Science University
More Information
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Additional Information:

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Responsible Party: Susan J. Hayflick, Professor & Chair, Molecular & Medical Genetics, Oregon Health and Science University
ClinicalTrials.gov Identifier: NCT02587858    
Other Study ID Numbers: 10832
First Posted: October 27, 2015    Key Record Dates
Last Update Posted: October 8, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Susan J. Hayflick, Oregon Health and Science University:
NBIA
PKAN
brain iron
Neurodegeneration with Brain Iron Accumulation
Hallervorden Spatz Syndrome
Pantothenate Kinase Associated Neurodegeneration
PLAN
 INAD
PLA2G6-associated neurodegeneration
BPAN
Beta-propeller Protein-associated neurodegenration
WDR45
PLA2G6
Additional relevant MeSH terms:
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Pantothenate Kinase-Associated Neurodegeneration
Nerve Degeneration
Pathologic Processes
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Neuroaxonal Dystrophies
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn