Clinical Trials on Fatty Acid Hydroxylase-associated Neurodegeneration (FAHN)

Total 9 results

  • LMU Klinikum
    Seventh Framework Programme; NBIA Alliance
    Recruiting
    TIRCON International NBIA Registry (TIRCON)
    Neurodegeneration With Brain Iron Accumulation (NBIA) | Pantothenate Kinase-associated Neurodegeneration (PKAN) | Aceruloplasminemia | Beta-Propeller Protein-Associated Neurodegeneration (BPAN) | Mitochondrial Membrane Protein Associated Neurodegeneration (MPAN) | Fatty Acid Hydroxylase-associated... and other conditions
    Canada, Czechia, Germany, Italy, Netherlands, Poland, Serbia, Spain
  • Fresenius Kabi
    Not yet recruiting
    Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment
    Malnutrition | Pediatric ALL | Essential Fatty Acid Deficiency | Parenteral Nutrition Associated Liver Disease (PNALD)
  • Fresenius Kabi
    Withdrawn
    Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
    Malnutrition | Pediatric ALL | Neurocognitive Deficit | Parenteral Nutrition Associated Liver Disease | Essential Fatty Acid Deficiency
  • University of Jena
    Jena University Hospital
    Completed
    Evaluating the Potential of the Diet as Disease Modifier in Amyotrophic Lateral Sclerosis (JERN_ALS) (JERN_ALS)
    Amyotrophic Lateral Sclerosis | Dietary Habits | Disorder of Fatty Acid Metabolism
    Germany
  • University Hospital, Bordeaux
    Recruiting
    Functional Tests to Resolve Unsolved Rare Diseases. Rares. (RID)
    Intellectual Disability | Cystic Fibrosis | Neurodegeneration With Brain Iron Accumulation (NBIA) | Congenital Heart Defect | Albinism | Rubinstein-Taybi Syndrome | Periventricular Nodular Heterotopia
    France
  • Centre Hospitalier Universitaire de Liege
    Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators
    Recruiting
    Baby Detect : Genomic Newborn Screening
    Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions
    Belgium
  • UK Kidney Association
    Recruiting
    National Registry of Rare Kidney Diseases (RaDaR)
    Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions
    United Kingdom
  • RTI International
    Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators
    Enrolling by invitation
    Early Check: Expanded Screening in Newborns
    Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions
    United States
  • Sanford Health
    National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators
    Recruiting
    Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)
    Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions
    United States, Australia

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