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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

Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

NCT04531189

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Jackson Memorial Hospital Miami Transplant Unit University of Miami Miller School of Medicine Miami, Florida, United States
  • Northwestern University Feinberg School of Medicine Chicago, Illinois, United States
  • University of Michigan Michigan Medicine Hepatology Clinic Taubman Center Ann Arbor, Michigan, United States
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Completed

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT01896102

Conditions
Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +22 more
Locations
  • Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center Los Angeles, California, United States
  • Boston Children's Hospital/Massachusetts General Hospital Boston, Massachusetts, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Medeos SRL Buenos Aires, Argentina
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Completed

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

NCT01016392

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Research Site Graz, Austria
  • Research Site Innsbruck, Austria
  • Research Site Salzburg, Austria
  • Research Site Vienna, Austria
View all 69 locations
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Completed

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

NCT01410890

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations
  • Investigational Site Number 840008 Valhalla, New York, United States
  • Investigational Site Number 840007 Cincinnati, Ohio, United States
  • Investigational Site Number 840005 Salt Lake City, Utah, United States
  • Investigational Site Number 840003 Fairfax, Virginia, United States
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Completed

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

NCT02292654

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Investigational Site Number 840001 New York, United States
  • Investigational Site Number 076001 Porto Alegre, Brazil
  • Investigational Site Number 250002 Bron, France
  • Investigational Site Number 276001 Mainz, Germany
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