Pantothenate Kinase-Associated NeurodegenerationNCT04182763
CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
A study testing CoA-Z in people with Pantothenate Kinase-Associated Neurodegeneration, run by Oregon Health and Science University at 1 site in United States.
Trial summary
The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product. We are hoping to find out if the study product is safe, what effects-good and bad-the study product causes, and whether the study product… Read more about this study
- Who is it for?
- Ages 3 months to 89 years
- Conditions the study is for
- Pantothenate Kinase-Associated Neurodegeneration
- What is being tested?
- CoA-Z
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 77 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Dec 2019to 1 Jan 2025
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 months to 89 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Has a diagnosis of PKAN confirmed by: a) genetic testing confirming 2 pathogenic or likely pathogenic mutations, or (b) typical findings on exam and brain MR imaging with only one pathogenic mutation +/- a second likely pathogenic or VOUS in PANK2, or (c) typical findings on exam and brain MR imaging with a single likely pathogenic or VOUS in PANK2, or (d) be a symptomatic sibling of a proband subject meeting a, b or c.
- Be between 3 months old and 89 years old.
- Be able to take study product by mouth or feeding tube.
- Be willing and able to complete study procedures / telephone visits / blood draws independently, OR have a caregiver / parent willing and able to assist with these tasks.
- Be enrolled or willing to enroll in the PKANready natural history study (eIRB 10832).
- Be resident in North America (US or Canada) for the duration of the trial.
Who cannot join
- Have had exposure to a putative PANK2 bypass therapeutic agent in the 30 days prior to screening.
- Be concurrently enrolled in another interventional clinical trial.
- Have concurrent medical or other condition expected to preclude completion of study procedures of confound the assessment of clinical and laboratory measures of safety.
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health & Science University
Portland, Oregon, 97239
About this study
Official title: A Phase 2 Study of a Vitamin Metabolite for PKAN
The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product. We are hoping to find out if the study product is safe, what effects-good and bad-the study product causes, and whether the study product changes certain measures of disease in PKAN.
- Being tested
- CoA-Z, Placebo
- Keywords
- Neurodegeneration with Brain Iron Accumulation, NBIA, PKAN
- 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.
CoA-ZOther
People with PKAN lack a chemical to process or metabolize a certain vitamin in the brain. CoA-Z is designed to bypass this metabolic defect that causes PKAN.
PlaceboOther
The placebo is a strawberry-flavored syrup that looks and tastes like CoA-Z but has no active CoA-Z in it.
| Study group | Role | What they receive |
|---|---|---|
| CoA-Z dose 1 | Experimental | 6 months of CoA-Z at the highest assigned dose followed by 18 months of CoA-Z at dose 2 Receives: Other: CoA-Z |
| CoA-Z dose 2 | Experimental | 6 months of CoA-Z at the medium assigned dose followed by 18 months of CoA-Z at dose 2 Receives: Other: CoA-Z |
| CoA-Z dose 3 | Experimental | 6 months of CoA-Z at the lowest assigned dose followed by 18 months of CoA-Z at dose 2 Receives: Other: CoA-Z |
| Placebo | Placebo comparator | 6 months of placebo, followed by 18 months of CoA-Z at dose 2 (medium dose) Receives: Other: CoA-Z; Other: Placebo |
| Open-label arm | Experimental | Up to 24 months of CoA-Z at dose 2 Receives: Other: CoA-Z |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0
Safety will be measured using the number of treatment-emergent adverse events (both AE and SAE) by treatment arm. Counts are adjusted for number of prescription medications at baseline as a measure of baseline disease severity.
Measured: 6 months following first dose in double-blind phase
Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Complete Blood Count.
Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Complete Blood Count. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.
Measured: 6-month randomized, double-blind, placebo-controlled phase
Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Comprehensive Metabolic Profile.
Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.
Measured: 6-month randomized, double-blind, placebo-controlled phase
Number of Participants Retained in Each Arm.
Tolerability will be assessed by measuring the number of participants retained in each arm over the course of the study.
Measured: 6 month randomized, double-blind, placebo-controlled period
Mean Percent of Study Product Consumed.
Tolerability will be assessed by adherence to the study product regimen arm at each follow-up time point.
Measured: 6-month randomized, double-blind, placebo-controlled phase
Secondary outcomes (1)
CoASY mRNA Expression
Average relative CoASY gene expression, measured as the ratio to baseline of 1 / (2^[COASY Ct - 18s Ct]), where Ct = cycle time and 18s is a housekeeping gene. Values >1 reflect higher expression.
Measured: Up to 6 months after first dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2019
- Study start4 Dec 2019
- Primary completion1 Sep 2024
- Study completion1 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pantothenate Kinase-Associated NeurodegenerationStudies of the same condition, open and under way.
- More trials run by Oregon Health and Science UniversityThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- An initial 6-month, dose-ranging, parallel-group, randomized, double-blind, placebo-controlled phase is followed by an 18-month, single-dose, open-label phase--these arms met enrollment goals. A direct-to-open-label arm of the study was opened to allow for additional enrollment.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 77 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Washington State University, Oregon State University, Spoonbill Foundation, Spoonbill
- Responsible party
- Principal investigator, Susan J. Hayflick, Oregon Health and Science University
- Investigators
- Penelope Hogarth, M.D., Principal investigator, Oregon Health and Science UniversitySusan J Hayflick, MD, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04182763
- Sponsor's protocol ID
- IRB00018782
- Other IDs
- R01HD097328
- Status last verified
- Sep 2025
- First submitted
- 24 Nov 2019
- Last updated
- 29 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD will be entered in a stand-alone repository that will allow for future sharing with other researchers. At this time we have not yet determined what IPD are to be shared.
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of a Vitamin Metabolite for PKAN
- ClinicalTrials.gov ID
- NCT04182763
- Acronym / sponsor ID
- IRB00018782
- Keywords
- Neurodegeneration with Brain Iron Accumulation, NBIA, PKAN
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 77 participants (actual)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Washington State University, Oregon State University, Spoonbill Foundation, Spoonbill
- Study start
- 4 Dec 2019 (actual)
- Primary completion
- 1 Sep 2024 (actual)
- Study completion
- 1 Jan 2025 (actual)
- First posted
- 2 Dec 2019 (actual)
- Results
- Posted 29 Oct 2025
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 how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product. We are hoping to find out if the study product is safe, what effects-good and bad-the study product causes, and whether the study product changes certain measures of disease in PKAN.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
- Model description
- An initial 6-month, dose-ranging, parallel-group, randomized, double-blind, placebo-controlled phase is followed by an 18-month, single-dose, open-label phase--these arms met enrollment goals. A direct-to-open-label arm of the study was opened to allow for additional enrollment.
| Arm | Type | Receives |
|---|---|---|
| CoA-Z dose 1 | Experimental | Other: CoA-Z6 months of CoA-Z at the highest assigned dose followed by 18 months of CoA-Z at dose 2 |
| CoA-Z dose 2 | Experimental | Other: CoA-Z6 months of CoA-Z at the medium assigned dose followed by 18 months of CoA-Z at dose 2 |
| CoA-Z dose 3 | Experimental | Other: CoA-Z6 months of CoA-Z at the lowest assigned dose followed by 18 months of CoA-Z at dose 2 |
| Placebo | Placebo comparator | Other: CoA-Z, Other: Placebo6 months of placebo, followed by 18 months of CoA-Z at dose 2 (medium dose) |
| Open-label arm | Experimental | Other: CoA-ZUp to 24 months of CoA-Z at dose 2 |
Who may qualify
- Ages
- 3 months to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Has a diagnosis of PKAN confirmed by: a) genetic testing confirming 2 pathogenic or likely pathogenic mutations, or (b) typical findings on exam and brain MR imaging with only one pathogenic mutation +/- a second likely pathogenic or VOUS in PANK2, or (c) typical findings on exam and brain MR imaging with a single likely pathogenic or VOUS in PANK2, or (d) be a symptomatic sibling of a proband subject meeting a, b or c.
- Be between 3 months old and 89 years old.
- Be able to take study product by mouth or feeding tube.
- Be willing and able to complete study procedures / telephone visits / blood draws independently, OR have a caregiver / parent willing and able to assist with these tasks.
- Be enrolled or willing to enroll in the PKANready natural history study (eIRB 10832).
Key exclusion criteria (3 listed)
- Have had exposure to a putative PANK2 bypass therapeutic agent in the 30 days prior to screening.
- Be concurrently enrolled in another interventional clinical trial.
- Have concurrent medical or other condition expected to preclude completion of study procedures of confound the assessment of clinical and laboratory measures of safety.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CoA-ZPeople with PKAN lack a chemical to process or metabolize a certain vitamin in the brain. CoA-Z is designed to bypass this metabolic defect that causes PKAN. | Other | CoA-Z dose 1, CoA-Z dose 2, CoA-Z dose 3, Open-label arm, Placebo |
| PlaceboThe placebo is a strawberry-flavored syrup that looks and tastes like CoA-Z but has no active CoA-Z in it. | Other | Placebo |
Primary outcome
Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0
Time frame: 6 months following first dose in double-blind phase
Safety will be measured using the number of treatment-emergent adverse events (both AE and SAE) by treatment arm. Counts are adjusted for number of prescription medications at baseline as a measure of baseline disease severity.
Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Complete Blood Count.
Time frame: 6-month randomized, double-blind, placebo-controlled phase
Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Complete Blood Count. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.
Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Comprehensive Metabolic Profile.
Time frame: 6-month randomized, double-blind, placebo-controlled phase
Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.
Number of Participants Retained in Each Arm.
Time frame: 6 month randomized, double-blind, placebo-controlled period
Tolerability will be assessed by measuring the number of participants retained in each arm over the course of the study.
Mean Percent of Study Product Consumed.
Time frame: 6-month randomized, double-blind, placebo-controlled phase
Tolerability will be assessed by adherence to the study product regimen arm at each follow-up time point.
1 secondary outcome
CoASY mRNA Expression
Time frame: Up to 6 months after first dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Trial information websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. IPD will be entered in a stand-alone repository that will allow for future sharing with other researchers. At this time we have not yet determined what IPD are to be shared.
Related trials for Pantothenate Kinase-Associated Neurodegeneration
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.
Penelope Hogarth, M.D. principal investigator
Oregon Health and Science University
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?
A study testing CoA-Z in people with Pantothenate Kinase-Associated Neurodegeneration, run by Oregon Health and Science University 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 6 inclusion and 3 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 Oct 2025. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 29 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 (time frame: 6 months following first dose in double-blind phase). The study lists 5 primary outcomes in all. 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 Oct 2025.