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Clinical trial condition

Metabolism, Inborn Errors

Explore clinical trials studying Metabolism, Inborn Errors. Study availability and eligibility vary by location and protocol.

2,496 public trials 457 recruiting studies
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Metabolism, Inborn Errors trial results

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
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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

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

NCT06079359

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hypophosphatasia, +4 more
Locations
  • Research Site Baltimore, Maryland, United States
  • Research Site Kansas City, Missouri, United States
  • Research Site Durham, North Carolina, United States
  • Research Site Nedlands, Australia
View all 32 locations
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Active, Not Recruiting

X-linked Hypophosphatemia Disease Monitoring Program

NCT03651505

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +27 more
Locations
  • Children's Hospital of Alabama Birmingham, Alabama, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Rady Children's Hospital San Diego San Diego, California, United States
  • University of California San Francisco San Francisco, California, United States
View all 38 locations
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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

Evaluation of Efficacy and Safety of AR882 and XOI Co-administration in Uricase Treatment Failed Patients

NCT07116746

Conditions
Arthritis, Arthritis, Gouty, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +18 more
Locations
  • Arthrosi Investigative Site (105) Margate, Florida, United States
  • Arthrosi Investigative Site (102) Miami, Florida, United States
  • Arthrosi Investigative Site (109) Tampa, Florida, United States
  • Arthrosi Investigative Site (106) Jackson, Mississippi, United States
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Active, Not Recruiting

Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients (AR882-301)

NCT06846515

Conditions
Arthritis, Arthritis, Gouty, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +14 more
Locations
  • Arthrosi Investigative Site (620) Birmingham, Alabama, United States
  • Arthrosi Investigative Site (616) Huntsville, Alabama, United States
  • Arthrosi Investigative Site (680) Peoria, Arizona, United States
  • Arthrosi Investigative Site (640) Phoenix, Arizona, United States
View all 83 locations
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Active, Not Recruiting

An Adapted Brazilian Cardioprotective Diet, Phytosterols and Krill Oil in Familial Hypercholesterolemia (DICA-FH)

NCT06331195

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypercholesterolemia, +9 more
Locations
  • Hcor São Paulo, Brazil
  • OCARA Belém, Brazil
  • Centro Oncológico de Roraima Boa Vista, Brazil
  • Instituto de Cardiologia e Transplantes do DF Brasília, Brazil
View all 24 locations
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