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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.

2,493 public trials 456 recruiting studies
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Metabolism, Inborn Errors trial results

Active, Not Recruiting

Amyloidosis TTR Flow Reserve Evaluation

NCT05103943

Conditions
Amyloid Cardiomyopathy, Amyloid Neuropathies, Amyloid Neuropathies, Familial, +13 more
Locations
  • CHU d'ANGERS Angers, France
  • CHU Caen Caen, France
  • CHU Orleans Orléans, France
  • CHRU de TOURS Tours, France
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Active, Not Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • University of Minnesota, Pediatrics Minneapolis, Minnesota, United States
  • Ospedale San Raffaele Milan, Italy
  • Princess Maxima Center Utrecht, Netherlands
  • UMC Utrecht Utrecht, Netherlands
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Active, Not Recruiting

A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

NCT05095727

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +6 more
Locations
  • University of Connecticut Health Center Farmington, Connecticut, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Duke University Medical Center Durham, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
View all 16 locations
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Active, Not Recruiting

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

NCT06031259

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations
  • M.A.G.I.C. Clinic Calgary, Alberta, Canada
  • University of British Columbia Vancouver, British Columbia, Canada
  • Hospital for Sick Children Toronto, Ontario, Canada
  • Hopital Femme Mere Enfant Bron, Auvergne-Rhône-Alpes, France
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Active, Not Recruiting

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Lysosomal Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Hospital de Clinicas de Porto Alegre (HCPA) Porto Alegre, Brazil
  • Hospital Quironsalud Zaragoza Zaragoza, Spain
  • Salford Royal Hospital Salford, United Kingdom
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Active, Not Recruiting

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT05164055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Investigational Site Number : 2500004 Bordeaux, France
  • Investigational Site Number : 2500005 Brest, France
  • Investigational Site Number : 2500008 Clermont-Ferrand, France
  • Investigational Site Number : 2500009 Lille, France
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Active, Not Recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • Wolfson Children's Hospital Jacksonville, Florida, United States
  • Woodruff Memorial Research Building Atlanta, Georgia, United States
  • St. Louis Children's Hospital St Louis, Missouri, United States
View all 17 locations
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