Not applicableEnrolling By InvitationNCT06912841What this trial is testingDeep Brain Stimulation (DBS) MatchMakerWho this might be right forTOR1APANK2HPRT1+14 more Boston Children's Hospital 500
Large-scale testing (Phase 3)Study completedNCT02174848What this trial is testingLong-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated NeurodegenerationWho this might be right forPantothenate Kinase-Associated Neurodegeneration ApoPharma 68
Not applicableLooking for participantsNCT05522374What this trial is testingTIRCON International NBIA RegistryWho this might be right forNeurodegeneration With Brain Iron Accumulation (NBIA)Pantothenate Kinase-associated Neurodegeneration (PKAN)Beta-Propeller Protein-Associated Neurodegeneration (BPAN)+8 more LMU Klinikum 2,000
Not applicableStudy completedNCT05615571What this trial is testingTesting of NBIA Genes: Analysis of Genetic Heterogeneity and Validation of Mitochondrial Markers for Assessing Causality of Sequence Variants.Who this might be right forNeurodegeneration With Brain Iron Accumulation (NBIA) University Hospital, Bordeaux 70
Large-scale testing (Phase 3)Ended earlyNCT03041116What this trial is testingEfficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN ParticipantsWho this might be right forPantothenate Kinase-Associated Neurodegeneration Travere Therapeutics, Inc. 84
Not applicableNo Longer AvailableNCT02635841What this trial is testingCompassionate Use of Deferiprone in Patients With PKANWho this might be right forPantothenate Kinase-Associated Neurodegeneration Chiesi Canada Corp
Not applicableLooking for participantsNCT06596746What this trial is testingNeurodegenerative Diseases Progression Markers (MARKERS-NDD)Who this might be right forNeurodegenerative DiseasesParkinson DiseaseSynucleinopathies+15 more Casa di Cura San Raffaele Cassino 600
Not applicableStudy completedNCT04182763What this trial is testingCoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)Who this might be right forPantothenate Kinase-Associated Neurodegeneration Oregon Health and Science University 77
Not applicableLooking for participantsNCT05696912What this trial is testingFunctional Tests to Resolve Unsolved Rare Diseases. Rares.Who this might be right forIntellectual DisabilityRubinstein-Taybi SyndromeCystic Fibrosis+4 more University Hospital, Bordeaux 50
Not applicableStudy completedNCT01838018What this trial is testingBrain Perfusion in Pantothenate Kinase-associated Neurodegeneration (PKAN)Who this might be right forPantothenate Kinase-associated Neurodegeneration (PKAN) Susan J. Hayflick 10
Not applicableUnknownNCT02587858What this trial is testingNBIAready: Online Collection of Natural History Patient-reported Outcome MeasuresWho this might be right forNeurodegeneration With Brain Iron Accumulation (NBIA) Susan J. Hayflick 300
Large-scale testing (Phase 3)Study completedNCT01741532What this trial is testingEfficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)Who this might be right forPantothenate Kinase-Associated Neurodegeneration ApoPharma 89
Not applicableUnknownNCT04319796What this trial is testingEuropean Registry on Rare Neurological DiseasesWho this might be right forRare Diseases University Hospital Tuebingen 5,000