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ClinicalTrials.gov public records Last synced May 21, 2026, 6:37 PM EDT

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Showing 1–7 of 7 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: PKAN Clear all
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
CoA-Z, Placebo
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
3 Months to 89 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 28, 2025 · Synced May 21, 2026, 6:37 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 8, 2026 · Synced May 21, 2026, 6:37 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Pantothenate Kinase-associated Neurodegeneration (PKAN)
Interventions
Not listed
Lead sponsor
Susan J. Hayflick
Other
Eligibility
7 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 28, 2018 · Synced May 21, 2026, 6:37 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
Deferiprone oral solution, Placebo
Drug
Lead sponsor
ApoPharma
Industry
Eligibility
4 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jul 4, 2019 · Synced May 21, 2026, 6:37 PM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
Fosmetpantotenate, Placebo
Drug
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
6 Years to 65 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
9
States / cities
Irvine, California • Washington D.C., District of Columbia • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2021 · Synced May 21, 2026, 6:37 PM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Neurodegeneration With Brain Iron Accumulation (NBIA)
Interventions
Not listed
Lead sponsor
Susan J. Hayflick
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 7, 2020 · Synced May 21, 2026, 6:37 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
Deferiprone oral solution
Drug
Lead sponsor
ApoPharma
Industry
Eligibility
5 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Aug 24, 2020 · Synced May 21, 2026, 6:37 PM EDT