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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.

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

Completed

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

NCT01898364

Conditions
Acid Maltase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +15 more
Locations
  • Investigational Site Number 840006 Phoenix, Arizona, United States
  • Investigational Site Number 840010 Jacksonville, Florida, United States
  • Investigational Site Number 840001 Kansas City, Kansas, United States
  • Investigational Site Number 840008 St Louis, Missouri, United States
View all 17 locations
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Completed

Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness

NCT02746718

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • CHU d'Angers Angers, France
  • Centre hospitalier d'Argenteuil Argenteuil, France
  • CHU de Brest Brest, France
  • CH d'Escartons Briançon, France
View all 21 locations
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Completed

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

NCT05186324

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • #02 Birmingham, Alabama, United States
  • #03 Atlanta, Georgia, United States
  • #04 Iowa City, Iowa, United States
  • #11 Grand Rapids, Michigan, United States
View Trial Details
Completed

A Prospective Clinical Study of Phenylketonuria (PKU)

NCT04452513

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +9 more
Locations
  • Children's Hosptial of Colorado Aurora, Colorado, United States
  • University of Florida Gainesville, Florida, United States
  • University of South Florida Tampa, Florida, United States
  • Emory University Hospital Atlanta, Georgia, United States
View all 14 locations
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Completed

Phenylalanine and Its Impact on Cognition

NCT03788343

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Department of Diabetes, Endocrinology, Nutritional Medicine and Metabolism (UDEM), Inselspital, Bern University Hospital Bern, Switzerland
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Completed

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT02534844

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +34 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Children's Hospital of Orange County Orange, California, United States
  • University of California San Francisco San Francisco, California, United States
  • University of Colorado Denver Aurora, Colorado, United States
View all 29 locations
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