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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

Completed

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

NCT05139316

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +8 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • University of Connecticut Health Center Farmington, Connecticut, United States
  • Mount Sinai The Bronx, New York, United States
View all 20 locations
View Trial Details
Completed

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

NCT03566017

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +29 more
Locations
  • UAB Medicine Birmingham, Alabama, United States
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • University of California San Diego La Jolla, California, United States
  • University of California Irvine Center Orange, California, United States
View all 30 locations
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Completed

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

NCT03970278

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +9 more
Locations
  • UCONN Health Farmington, Connecticut, United States
  • Michigan Medicine University of Michigan Ann Arbor, Michigan, United States
  • University of Texas Health Science Center at Houston Houston, Texas, United States
  • Montreal Children Hospital, McGill University Health Centre Montreal, Quebec, Canada
View Trial Details
Completed

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

NCT06164951

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +16 more
Locations
  • QED Investigative Site San Francisco, California, United States
  • QED Investigative Site Aurora, Colorado, United States
  • QED Investigative Site Baltimore, Maryland, United States
  • QED Investigative Site Columbia, Missouri, United States
View all 27 locations
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Completed

Early Detection of Fabry Disease

NCT05106764

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Universitätsklinikum Erlangen Kinder- und Jugendklinik Erlangen, Germany
  • Universitätsklinikum Erlangen Neurologische Klinik Erlangen, Germany
  • Universitätsklinikum Giessen Giessen, Germany
View Trial Details

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