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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 Treatment Study of Mucopolysaccharidosis Type IIIB

NCT02754076

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
Locations
  • Children's Hospital Oakland Oakland, California, United States
  • Fundación Cardio Infantil - Instituto de Cardiología Bogotá, Colombia
  • University Medical Center Hamburg Eppendorf, Department of Pediatrics Hamburg, Germany
  • Hospital Clinico Universitario de Santiago Santiago de Compostela, Spain
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Completed

A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis

NCT01681953

Conditions
Alpha-Mannosidosis, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +7 more
Locations
  • Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9 Copenhagen, Denmark
  • Hôpital Femme Mère Enfant, Lyon, 59 boulevard Pinel Bron, France
  • Hôpital Trousseau, Service de neuropédiatrie, Centre Référence des Maladies Lysosomales, 26 avenue du Docteur Arnold Netter Paris, France
  • Universitätsmedizin Mainz, Zentrum für Kinder- und Jugendmedizin, Langenbeckstrasse 1 Mainz, Germany
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Completed

The Natural History of Alpha-Mannosidosis

NCT00498420

Conditions
Alpha Mannosidosis, Bone Diseases, Bone Diseases, Developmental, +15 more
Locations
  • Department of Pediatrics, Charles University Prague, Czechia
  • University of Mainz Mainz, Germany
  • Department of Medicine, University of Tromsoe Tromsø, Norway
  • Willink Biochemical Genetics Unit,. Royal Manchester Children's Hospital Manchester, United Kingdom
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Completed

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Orange County Orange, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Baylor College of Medicine Houston, Texas, United States
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Completed

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

NCT02230566

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations
  • Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Orange County Orange, California, United States
  • Miami Children's Hospital Miami, Florida, United States
  • University of Minnesota Minneapolis, Minnesota, United States
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