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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

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California Irvine, Department of Neurology Orange, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Utah, Division of Medical Genetics Salt Lake City, Utah, United States
  • Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility Newcastle upon Tyne, United Kingdom
View Trial Details
Active, Not Recruiting

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • University of California Los Angeles, California, United States
  • University of Colorado Aurora, Colorado, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University Hospitals Cleveland Medical Center Cleveland, Ohio, United States
View all 16 locations
View Trial Details
Active, Not Recruiting

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • The Children's Hospital Colorado Aurora, Colorado, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Icahn School of Medicine New York, United States
  • University Hospital Cleveland Medical Center/Case Western Reserve University Cleveland, Ohio, United States
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Active, Not Recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

NCT06052800

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Investigational Site Number : 1580004 Taichung, Taiwan
  • Investigational Site Number : 1580005 Tainan, Taiwan
  • Investigational Site Number : 1580001 Taipei, Taiwan
  • Investigational Site Number : 1580003 Taipei, Taiwan
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Active, Not Recruiting

Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial

NCT07226622

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • University of California Los Angeles Medical Center Los Angeles, California, United States
  • University of California Davis Health Sacramento, California, United States
  • Yale New Haven Hospital New Haven, Connecticut, United States
  • Northwestern University Chicago, Illinois, United States
View Trial Details
Active, Not Recruiting

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

NCT06095713

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Fabry disease center Berlin - Charité - Universitätsmedizin Berlin Berlin, Germany
  • Fabry disease center Cologne, Universitätsklinikum Köln Cologne, Germany
  • Fabry disease center Hamburg, Universitätsklinikum Hamburg Hamburg, Germany
  • Fabry disease center Hannover, Universitätsklinikum Hannover Hanover, Germany
View Trial Details
Active, Not Recruiting

Liver Disease in Urea Cycle Disorders

NCT04612764

Conditions
ARGI Deficiency, ASL Deficiency, ASS Deficiency, +22 more
Locations
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Baylor College of Medicine Houston, Texas, United States
View Trial Details

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