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Trial record 1 of 7 for:    Neurodegeneration with Brain Iron Accumulation (NBIA)
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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 : May 28, 2018
Sponsor:
Collaborator:
Oregon Health and Science University
Information provided by (Responsible Party):
Susan J. Hayflick, Oregon Health and Science University

Tracking Information
First Submitted Date October 23, 2015
First Posted Date October 27, 2015
Last Update Posted Date May 28, 2018
Study Start Date April 2015
Estimated Primary Completion Date April 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 23, 2015)
Document the natural history of NBIA and identify new markers of disease progression. [ Time Frame: 5 years ]
The unit of analyses will be the data collected on NBIA milestones and patient-reported outcome measures. Descriptive statistics 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. Descriptive statistics will be used to characterize the natural history of NBIA and identify areas in which individuals with NBIA differ from the general population.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT02587858 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
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 be (1) collecting information about how symptoms and findings in NBIA change over time and (2) identifying 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 years to electronically complete a set of rating scales as related to their NBIA disorder.

Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
BPAN participants will be asked for blood and saliva samples.
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.
Condition Neurodegeneration With Brain Iron Accumulation (NBIA)
Intervention Not Provided
Study Groups/Cohorts
  • PKAN
    This group consists of individuals diagnosed with PKAN using a combination of MRI and PANK2 gene sequencing.
  • PLAN
    This group consists of individuals diagnosed with PLAN from PLA2G6 gene sequencing.
  • BPAN
    This group consists of individuals diagnosed with BPAN from WDR45 gene sequencing.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 23, 2015)
300
Original Estimated Enrollment Same as current
Estimated Study Completion Date April 2025
Estimated Primary Completion Date April 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Diagnosis of PKAN, PLAN or BPAN confirmed gene testing.
  • Ability to access a computer with internet services approximately once every 6 months for up to 5 years to enter data.

Exclusion Criteria:

  • Individuals who are not fluent in reading and communicating in English.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Alison C Freed, BA 503 494 6838 freeal@ohsu.edu
Contact: Allison M Gregory, MS, CGC 503 494 4344 gregorya@ohsu.edu
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02587858
Other Study ID Numbers 10832
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Susan J. Hayflick, Oregon Health and Science University
Study Sponsor Susan J. Hayflick
Collaborators Oregon Health and Science University
Investigators
Principal Investigator: Susan J Hayflick, MD Oregon Health and Science University
PRS Account Oregon Health and Science University
Verification Date May 2018