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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 Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

NCT03681184

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, +13 more
Locations
  • Clinical Trial Site San Diego, California, United States
  • Clinical Trial Site Jacksonville, Florida, United States
  • Clinical Trial Site Rochester, Minnesota, United States
  • Clinical Trial Site New York, United States
View all 17 locations
View Trial Details
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

Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients

NCT06406153

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine Mashhad, Iran
  • Growth and Development Research Center, Childrens Medical Center Tehran, Iran
  • Loghman Hospital Tehran, Iran
  • Mofid Childrens Hospital Tehran, Iran
View Trial Details
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

A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH

NCT04819490

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • Royal Hospital for Sick Children Edinburgh, United Kingdom
  • NHS Greater Glasgow & Clyde- South Glasgow University Hosp Division Glasgow, United Kingdom
  • Leeds Teaching Hospitals NHS Trust, Trust Headquarters, St. James's University Hospital Leeds, United Kingdom
  • Evelina Childrens Hospital London, United Kingdom
View Trial Details
Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia

NCT04045145

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +16 more
Locations
  • Neurocrine Clinical Site San Diego, California, United States
  • Neurocrine Clinical Site Aurora, Colorado, United States
  • Neurocrine Clinical Site Ann Arbor, Michigan, United States
  • Neurocrine Clinical Site Minneapolis, Minnesota, United States
View Trial Details

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