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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

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care

NCT02320084

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Swedish Orphan Biovitrum Investigational Site Innsbruck, Austria
  • Swedish Orphan Biovitrum Investigational Site Vienna, Austria
  • Swedish Orphan Biovitrum Investigational Site Antwerp, Belgium
  • Swedish Orphan Biovitrum Investigational Site Brussels, Belgium
View all 77 locations
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Completed

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

NCT01257737

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • UCLA Pediatrics/Genetics Los Angeles, California, United States
  • Stanford University School of Medicine Palo Alto, California, United States
  • Denver Children's Hospital Aurora, Colorado, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
View all 16 locations
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Completed

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

NCT03425539

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 College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation Gainesville, Florida, United States
View all 49 locations
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Completed

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

NCT04442347

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • University of Florida Gainesville, Florida, United States
  • M Health Fairview Masonic Children's Hospital Minneapolis, Minnesota, United States
  • The Mount Sinai Hospital New York, United States
  • Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
View Trial Details
Completed

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

NCT00947544

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • UCLA Los Angeles, California, United States
  • The George Washington DC Children's National Medical Center Washington D.C., District of Columbia, United States
  • Mount Sinai School of Medicine New York, United States
  • University of Pittsburgh Pittsburgh, Pennsylvania, United States
View Trial Details
Completed

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

NCT01347073

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • UCLA Pediatrics/Genetics Los Angeles, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Maine Medical Center Portland, Maine, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View Trial Details
Completed

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

NCT00947297

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • Long Beach Memorial Long Beach, California, United States
  • UCLA Los Angeles, California, United States
  • Stanford University Stanford, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 22 locations
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