Not applicableLooking for participantsNCT03333200What this trial is testingLongitudinal Study of Neurodegenerative DisordersWho this might be right forMLDKrabbe DiseaseALD+24 more University of Pittsburgh 1,500
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 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)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 applicableUnknownNCT05696912What 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 applicableUnknownNCT04319796What this trial is testingEuropean Registry on Rare Neurological DiseasesWho this might be right forRare Diseases University Hospital Tuebingen 5,000
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 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 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
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 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 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