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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.

2,499 public trials 456 recruiting studies
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Metabolism, Inborn Errors trial results

Completed

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004704

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Investigational Site Number 840001 New York, United States
  • Investigational Site Number 056001 Leuven, Belgium
  • Investigational Site Number 076001 Porto Alegre, Brazil
  • Investigational Site Number 250002 Bron, France
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Completed

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

NCT03335488

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • University of Florida (UF) - Shands Hospital Gainesville, Florida, United States
  • Mount Sinai School of Medicine New York, United States
  • University Hospitals Case Medical Center Cleveland, Ohio, United States
  • Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania, United States
View Trial Details
Completed

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

NCT02246218

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • Kaiser Permanente Regional Metabolic Center Los Angeles, California, United States
  • Stanford Center for Clinical & Translational Research & Education Palo Alto, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Shands at University of Florida Gainesville, Florida, United States
View all 17 locations
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Completed

Pegloticase and Methotrexate Co-administered in Participants With Uncontrolled Gout Who Previously Failed Pegloticase Monotherapy

NCT04772313

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +10 more
Locations
  • University of Alabama at Birmingham (UAB) - Center for Education & Research on Therapeutics of Musculoskeletal Disorders Birmingham, Alabama, United States
  • Arizona Arthritis and Rheumatology Associates Flagstaff, Arizona, United States
  • Arizona Arthritis and Rheumatology Associates Glendale, Arizona, United States
  • Arizona Arthritis and Rheumatology Associates Mesa, Arizona, United States
View all 15 locations
View Trial Details
Completed

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

NCT03860935

Conditions
Alzheimer Disease, Amyloid Cardiomyopathy, Amyloid Neuropathies, +25 more
Locations
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • Pacific Heart Institute Santa Monica, California, United States
  • University of Colorado Hospital - Anschutz Medical Campus Aurora, Colorado, United States
  • Yale School of Medicine New Haven, Connecticut, United States
View all 104 locations
View Trial Details
Completed

Study of Pegloticase (KRYSTEXXA®) Plus Methotrexate in Patients With Uncontrolled Gout

NCT03635957

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +9 more
Locations
  • Orthopedic Physicians Alaska Anchorage, Alaska, United States
  • Arizona Arthritis & Rheumatology -West Valley Glendale, Arizona, United States
  • Arizona Arthritis & Rheumatology -East Valley Mesa, Arizona, United States
  • Avail Clinical Research DeLand, Florida, United States
View Trial Details
Completed

Study of KRYSTEXXA® (Pegloticase) Plus Methotrexate in Participants With Uncontrolled Gout

NCT03994731

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +9 more
Locations
  • University of Alabama Birmingham, Alabama, United States
  • Orthopedic Physicians Alaska Anchorage, Alaska, United States
  • Arizona Arthritis and Rheumatology Research, PLLC - Flagstaff Flagstaff, Arizona, United States
  • Arizona Arthritis and Rheumatology Research, PLLC-West Glendale, Arizona, United States
View all 60 locations
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