Neurodegeneration With Brain Iron Accumulation (NBIA)NCT02587858
NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
An observational study of Neurodegeneration With Brain Iron Accumulation (NBIA), run by Susan J. Hayflick at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Neurodegeneration With Brain Iron Accumulation (NBIA)
- How many people
- 300 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2015to Apr 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- Individuals who are not fluent in reading and communicating in English.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Oregon Health & Science University
Portland, Oregon, 97239-3098
Alison C Freed, BA, 503-494-6838, freeal@ohsu.edu
Allison M Gregory, MS, CGC, 503 494 4344, gregorya@ohsu.edu
About this study
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.
- Keywords
- 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
- Medical subject headings
- Pantothenate Kinase-Associated Neurodegeneration, Neuroaxonal Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Document the natural history of NBIA and identify new markers of disease progression.
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.
Measured: 5-10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2015 (estimated)
- Study startApr 2015
- Primary completionApr 2025 (estimated)
- Study completionApr 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurodegeneration With Brain Iron Accumulation (NBIA)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Susan J. Hayflick
- Sponsor type
- University, hospital or other
- Collaborators
- Oregon Health and Science University
- Responsible party
- Sponsor-investigator, Susan J. Hayflick, Oregon Health and Science University
- Investigators
- Susan J Hayflick, MD, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02587858
- Sponsor's protocol ID
- 10832
- Status last verified
- Oct 2020
- First submitted
- 23 Oct 2015
- Last updated
- 8 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- NBIAready: Online Collection of Natural History Patient-reported Outcome Measures
- ClinicalTrials.gov ID
- NCT02587858
- Acronym / sponsor ID
- 10832
- Keywords
- NBIA, PKAN, brain iron, Neurodegeneration with Brain Iron Accumulation, Hallervorden Spatz Syndrome, Pantothenate Kinase Associated Neurodegeneration, PLAN, INAD
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Susan J. HayflickUniversity, hospital or other
- Collaborators
- Oregon Health and Science University
- Study start
- Apr 2015
- Primary completion
- Apr 2025 (estimated)
- Study completion
- Apr 2025 (estimated)
- First posted
- 27 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Individuals who are not fluent in reading and communicating in English.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2020.
- Official website of the OHSU NBIA research groupLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Susan J Hayflick, MD principal investigator
Oregon Health and Science University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurodegeneration With Brain Iron Accumulation (NBIA), run by Susan J. Hayflick at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Alison C Freed, BA (503 494 6838, freeal@ohsu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Document the natural history of NBIA and identify new markers of disease progression. (time frame: 5-10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2020.