Clinical Trials on Metachromatic Leukodystrophy

Total 61 results

  • CENTOGENE GmbH Rostock
    Withdrawn
    Biomarker for Krabbe Disease (BioKrabbe) (BIOKRABBE)
    Krabbe Disease
    Egypt, Germany, India, Sri Lanka
  • Forge Biologics, Inc
    Not yet recruiting
    Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
    Krabbe Disease
    United States
  • University of Pittsburgh
    National Institute of Neurological Disorders and Stroke (NINDS)
    Active, not recruiting
    Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease
    Krabbe Disease
    United States
  • Forge Biologics, Inc
    Active, not recruiting
    Gene Transfer Clinical Trial for Krabbe Disease (RESKUE)
    Krabbe Disease
    United States
  • Hugo W. Moser Research Institute at Kennedy Krieger...
    Recruiting
    Home Exercise for Individuals With Neurodegenerative Disease
    Neurodegenerative Diseases | Ataxia | Cerebellar Ataxia | Leukodystrophy | Adrenomyeloneuropathy | LBSL | Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation | Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)
    United States
  • University of Pittsburgh
    Recruiting
    Longitudinal Study of Neurodegenerative Disorders
    Lysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditions
    United States
  • University of Chicago
    National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators
    Completed
    Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
    Krabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)
    United States
  • Masonic Cancer Center, University of Minnesota
    Terminated
    Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
    Sphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type B
    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
  • 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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