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Clinical trial condition

Brain Diseases, Metabolic, Inborn

Explore clinical trials studying Brain Diseases, Metabolic, Inborn. Study availability and eligibility vary by location and protocol.

948 public trials 197 recruiting studies
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Brain Diseases, Metabolic, Inborn trial results

Active, Not Recruiting

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +32 more
Locations
  • Emory University Atlanta, Georgia, United States
  • The Cleveland Clinic Cleveland, Ohio, United States
  • Lysosomal and Rare Disorders Research and Treatment Center, Inc Fairfax, Virginia, United States
  • Royal Melbourne Hospital Parkville, Victoria, Australia
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Active, Not Recruiting

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

NCT03737214

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama at Birmingham - Nephrology Research Clinic Birmingham, Alabama, United States
  • University of California Irvine Irvine, California, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Florida Clinical and Translational Science Institute, UF Clinical Research Center Gainesville, Florida, United States
View all 42 locations
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Active, Not Recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
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Active, Not Recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475

Conditions
Beta-Galactosidase-1 (GLB1) Deficiency, Brain Diseases, Brain Diseases, Metabolic, +23 more
Locations
  • Benioff Children's Hospital Oakland, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Children's Hospital at St. Peter's University Hospital New Brunswick, New Jersey, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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Active, Not Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama -The Kirklin Clinic- Site Number : 8400010 Birmingham, Alabama, United States
  • University of California Los Angeles Medical Center- Site Number : 8400008 Los Angeles, California, United States
  • Emory University School of Medicine - Atlanta- Site Number : 8400009 Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005 Chicago, Illinois, United States
View all 54 locations
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Active, Not Recruiting

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

NCT04848779

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Advanced Medical Genetics- Site Number : 8400002 Hawthorne, New York, United States
  • Duke University Medical Center- Site Number : 8400004 Durham, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center- Site Number : 8400001 Cincinnati, Ohio, United States
  • Le Bonheur Children's Hospital- Site Number : 8400005 Memphis, Tennessee, United States
View all 15 locations
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Active, Not Recruiting

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

NCT05166161

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Children's Hospital Colorado Aurora, Colorado, United States
  • UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism Gainesville, Florida, United States
  • Indiana University School of Medicine Department of Medical and Molecular Genetics Indianapolis, Indiana, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View all 46 locations
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Active, Not Recruiting

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

NCT04884815

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • Stanford University Redwood City, California, United States
  • University of California Davis Sacramento, California, United States
  • Northwestern University Chicago, Illinois, United States
View all 16 locations
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