Pantothenate Kinase-associated Neurodegeneration (PKAN)NCT01838018
Brain Perfusion in Pantothenate Kinase-associated Neurodegeneration (PKAN)
An observational study of Pantothenate Kinase-associated Neurodegeneration (PKAN), run by Susan J. Hayflick at 1 site in United States.
Trial summary
The purpose of this study is to learn whether blood flow in the brain is normal in people with pantothenate kinase-associated neurodegeneration (PKAN). Specifically, preliminary data suggest a region of the brain called the globus pallidus (GP), a key region affected by PKAN, may have reduced blood flow. Read more about this study
- Who is it for?
- Ages 7 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Pantothenate Kinase-associated Neurodegeneration (PKAN)
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2013to Jan 2018
- 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 7 to 75 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: The PKAN group consists of individuals in the United States diagnosed with PKAN.
Who may be able to join
- diagnosis of PKAN confirmed by MRI and gene testing
- able to undergo MRI for approximately 1 hour without sedation
Who cannot join
- deep brain stimulators or other conditions that warrant avoidance of a strong magnetic field
- previous reaction to gadolinium (Gd) contrast agent
- acute or chronic kidney dysfunction
- pregnant and/or nursing at time of study
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health & Science University
Portland, Oregon, 97239
About this study
Official title: Brain Perfusion in Pantothenate Kinase-Associated Neurodegeneration (PKAN)
The purpose of this study is to learn whether blood flow in the brain is normal in people with pantothenate kinase-associated neurodegeneration (PKAN). Specifically, preliminary data suggest a region of the brain called the globus pallidus (GP), a key region affected by PKAN, may have reduced blood flow. Standard MRI and perfusion scanning techniques will be used to learn about cerebral blood flow in the globus pallidus and compare it to blood flow in other brain regions as well as to healthy controls.
- Keywords
- PKAN, brain iron, NBIA, neurodegeneration with brain iron accumulation, Hallervorden Spatz Syndrome
- Medical subject headings
- Pantothenate Kinase-Associated Neurodegeneration
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 and PANK2 gene sequencing. | |
| Healthy volunteers | This is a control group of healthy volunteers, matched with the PKAN group for age and sex. |
What is being measured
The primary outcome is the main question the study is designed to answer.
ASL-derived cerebral blood flow(CBF)
The unit of analyses will be the subject and the primary comparison will be the globus pallidus CBF between PKAN and healthy controls.
Measured: Single MRI
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2013 (estimated)
- Study startApr 2013
- Primary completionOct 2017
- Study completionJan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pantothenate Kinase-associated Neurodegeneration (PKAN)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
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Susan J. Hayflick
- Sponsor type
- University, hospital or other
- Collaborators
- Oregon Health and Science University, Oregon Clinical and Translational Research Institute
- 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
- NCT01838018
- Sponsor's protocol ID
- 8777
- Status last verified
- May 2018
- First submitted
- 18 Apr 2013
- Last updated
- 29 May 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Brain Perfusion in Pantothenate Kinase-Associated Neurodegeneration (PKAN)
- ClinicalTrials.gov ID
- NCT01838018
- Acronym / sponsor ID
- 8777
- Keywords
- PKAN, brain iron, NBIA, neurodegeneration with brain iron accumulation, Hallervorden Spatz Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Susan J. HayflickUniversity, hospital or other
- Collaborators
- Oregon Health and Science University, Oregon Clinical and Translational Research Institute
- Study start
- Apr 2013 (actual)
- Primary completion
- Oct 2017 (actual)
- Study completion
- Jan 2018 (actual)
- First posted
- 23 Apr 2013 (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 whether blood flow in the brain is normal in people with pantothenate kinase-associated neurodegeneration (PKAN). Specifically, preliminary data suggest a region of the brain called the globus pallidus (GP), a key region affected by PKAN, may have reduced blood flow. Standard MRI and perfusion scanning techniques will be used to learn about cerebral blood flow in the globus pallidus and compare it to blood flow in other brain regions as well as to healthy controls.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PKAN | – | –This group consists of individuals diagnosed with PKAN using a combination of MRI and PANK2 gene sequencing. |
| Healthy volunteers | – | –This is a control group of healthy volunteers, matched with the PKAN group for age and sex. |
Who may qualify
- Ages
- 7 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The PKAN group consists of individuals in the United States diagnosed with PKAN.
Key inclusion criteria (2 listed)
- diagnosis of PKAN confirmed by MRI and gene testing
- able to undergo MRI for approximately 1 hour without sedation
Key exclusion criteria (4 listed)
- deep brain stimulators or other conditions that warrant avoidance of a strong magnetic field
- previous reaction to gadolinium (Gd) contrast agent
- acute or chronic kidney dysfunction
- pregnant and/or nursing at time of study
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
ASL-derived cerebral blood flow(CBF)
Time frame: Single MRI
The unit of analyses will be the subject and the primary comparison will be the globus pallidus CBF between PKAN and healthy controls.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2018.
- Related InfoLink provided by the sponsor.
Related trials for Pantothenate Kinase-associated Neurodegeneration (PKAN)
Not yet recruiting
Brain Iron in Beta-Propeller Protein Associated Neurodegeneration (BPAN)
Neurodegeneration With Brain Iron Accumulation (NBIA), Iron Metabolism Disorders
RecruitingObservational study
Neurodegenerative Diseases Progression Markers (MARKERS-NDD)
Neurodegenerative Diseases, Parkinson Disease
RecruitingObservational study
TIRCON International NBIA Registry
Neurodegeneration With Brain Iron Accumulation (NBIA), Pantothenate Kinase-associated Neurodegeneration (PKAN)
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 Pantothenate Kinase-associated Neurodegeneration (PKAN), 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 4 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: finished, as of the sponsor's last update on 29 May 2018. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ASL-derived cerebral blood flow(CBF) (time frame: Single MRI). 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 29 May 2018.