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Clinical trial condition

Sphingolipidoses

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

390 public trials 81 recruiting studies
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Sphingolipidoses trial results

Completed

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

NCT04041102

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
  • Montreal Children's Hospital Research Institute - McGill University Montreal, Quebec, Canada
View Trial Details
Completed

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

NCT06019728

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • UCLA Medical Center Site Number : 1003 Santa Monica, California, United States
  • Emory University School of Medicine Site Number : 1005 Atlanta, Georgia, United States
  • Infusion Associates Site Number : 1001 Grand Rapids, Michigan, United States
  • Metropolitan Hospital Center Site Number : 1004 New York, United States
View Trial Details
Completed

Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies

NCT05409846

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Centro Hospitalar Universitário de Coimbra Coimbra, Portugal
  • Hospital da Luz, Lisboa Lisbon, Portugal
  • Centro Hospitalar Universitário Lisboa Norte, EPE., Hospital de Santa Maria Lisbon, Portugal
  • Hospital Pedro Hispano (Unidade Local de Saúde Matosinhos) Matosinhos Municipality, Portugal
View Trial Details
Completed

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Mayo Clinic Rochester Rochester, Minnesota, United States
  • Hospital Pequeno Principe Curitiba, Brazil
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
View all 13 locations
View Trial Details
Completed

A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

NCT04845958

Conditions
Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +25 more
Locations
  • Investigational Site Number 2500036 Amiens, France
  • Investigational Site Number 2500014 Angers, France
  • Investigational Site Number 2500034 Argenteuil, France
  • Investigational Site Number 2500039 Bayonne, France
View all 40 locations
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