Clinical Trials on Carenza di proteine ​​trifunzionali mitocondriali

Total 8 results

  • Oregon Health and Science University
    National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Oregon...
    Terminated
    Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated Neuropathy
    Peripheral Neuropathy | Mitochondrial Trifunctional Protein Deficiency
    United States
  • Oregon Health and Science University
    University of Pittsburgh
    Completed
    Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder (Triheptanoin)
    Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase 2 (CPT2) Deficiency | Mitochondrial Trifunctional Protein (TFP) Deficiency | Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency
    United States
  • 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
  • McGill University
    Canadian Institutes of Health Research (CIHR)
    Completed
    Exogenous Ketosis During Bed Rest in Older Adults (KBR)
    Muscle Atrophy | Cognitive Function | Muscle Protein Synthesis | Insulin Sensitivity | Muscle Strength | Functional Capacity | Physical Inactivity | Mitochondrial Function
    Canada
  • Jerry Vockley, MD, PhD
    Ultragenyx Pharmaceutical Inc
    No longer available
    Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
    Barth Syndrome | Mitochondrial Trifunctional Protein Deficiency | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogen Storage Disorders | Pyruvate Carboxylase Deficiency... and other conditions
    United States
  • 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
  • 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

Sponsors and Collaborators

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CROs in Bangladesh

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