Vitamin compound studied for rare brain disease PKAN
NCT ID NCT04182763
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looked at how people with PKAN, a rare genetic brain disorder, respond to a special vitamin metabolite. Researchers wanted to see if it is safe and if it changes any signs of the disease. 77 people aged 3 months to 89 years took part.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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77 people
The number who actually took part.
- Started
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Dec 2019
- Finished
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Jan 2025
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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3 months to 89 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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. Exclusion Criteria: * 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Oregon Health & Science University
Portland, Oregon, 97239, United States