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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 Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

NCT04537377

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • UC Davis Medical Center Sacramento, California, United States
  • Yale University School of Medecine New Haven, Connecticut, United States
  • Advent Health Orlando, Florida, United States
  • University of Michigan Health System Ann Arbor, Michigan, United States
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Active, Not Recruiting

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Lysosomal Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Hospital de Clinicas de Porto Alegre (HCPA) Porto Alegre, Brazil
  • Hospital Quironsalud Zaragoza Zaragoza, Spain
  • Salford Royal Hospital Salford, United Kingdom
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Active, Not Recruiting

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT05164055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Investigational Site Number : 2500004 Bordeaux, France
  • Investigational Site Number : 2500005 Brest, France
  • Investigational Site Number : 2500008 Clermont-Ferrand, France
  • Investigational Site Number : 2500009 Lille, France
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Active, Not Recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • Wolfson Children's Hospital Jacksonville, Florida, United States
  • Woodruff Memorial Research Building Atlanta, Georgia, United States
  • St. Louis Children's Hospital St Louis, Missouri, United States
View all 17 locations
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Active, Not Recruiting

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • University of California San Francisco- Site Number : 8400007 San Francisco, California, United States
  • Children's Hospital IMD Clinic- Site Number : 8400015 Aurora, Colorado, United States
  • University of Florida-Genetics- Site Number : 8400010 Gainesville, Florida, United States
  • UHCMC- Site Number : 8400014 Cleveland, Ohio, United States
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