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Sphingolipidoses

Clinical trial condition

Sphingolipidoses

Explore clinical trials studying Sphingolipidoses. Study availability and eligibility vary by location and protocol.

394 public trials 77 recruiting studies

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Sphingolipidoses trial results

Completed

A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

NCT00446550

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations: Beverly Hills, California, United States

Coral Springs, Florida, United States

Decatur, Georgia, United States

Iowa City, Iowa, United States

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Completed

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations: Coral Springs, Florida, United States

Decatur, Georgia, United States

Boston, Massachusetts, United States

London, United Kingdom

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Completed

A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease

NCT02930655

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Investigator Site Würzburg, Germany

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Completed

The Natural History of Infantile Globoid Cell Leukodystrophy

NCT00983879

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Children's Hospital of Pittsburgh, 4401 One Children's Hospital Drive,4401 Penn Avenue Pittsburgh, Pennsylvania, United States

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Completed

Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1

NCT02124083

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +21 more
Locations: National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States

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Completed

Fabry Screening Study

NCT01019629

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Baylor Institute of Metabolic Disease Dallas, Texas, United States

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Completed

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
Locations: Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States

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Completed

Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease

NCT02995993

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Ruhruniversität Bochum, Klinik für Kinder- und Jugendmedizin im St. Josef-Hospital im Katholischen Klinikum Bochum Bochum, Germany

Universitätsmedizin Mainz, Zentrum für Kinder- und Jugendmedizin Mainz, Germany

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Completed

Screening for Fabry Disease in a Pediatric Population at Risk

NCT02152189

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +21 more
Locations: Zentrum für Kinder- und Jugendmedizin Heidelberg, Germany

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Completed

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

NCT01626092

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +50 more
Locations: Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States

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Sphingolipidoses

Sphingolipidoses trial results

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