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Clinical trial condition

Lysosomal Storage Diseases

Explore clinical trials studying Lysosomal Storage Diseases. Study availability and eligibility vary by location and protocol.

710 public trials 130 recruiting studies
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Lysosomal Storage Diseases trial results

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

Natural History of Atypical Morquio A Disease

NCT03204370

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Rheumatology department - Bordeaux University Hospital Bordeaux, Aquitaine, France
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

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
View Trial Details
Completed

China Post-marketing Surveillance (PMS) Study of Aldurazyme®

NCT05134571

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
Locations
  • Investigational Site Number :1560003 Beijing, China
  • Investigational Site Number :1560002 Beijing, China
  • Investigational Site Number :1560004 Guangzhou, China
  • Investigational Site Number :1560006 Hangzhou, China
View Trial Details
Completed

Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ

NCT04656600

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Investigational Site Number : 107 Beijing, China
  • Investigational Site Number : 101 Beijing, China
  • Investigational Site Number : 104 Chengdu, China
  • Investigational Site Number : 102 Guangzhou, China
View Trial Details
Completed

China Post-marketing Surveillance (PMS) Study of Fabrazyme®

NCT05054387

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Investigational Site Number :1560003 Beijing, China
  • Investigational Site Number :1560002 Beijing, China
  • Investigational Site Number :1560001 Shanghai, China
  • Investigational Site Number :1560004 Shanghai, China
View Trial Details
Completed

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

NCT03729362

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Neuromuscular Research Center Phoenix, Arizona, United States
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • University of California, Irvine Irvine, California, United States
  • UF Helath: University of Florida Clinical Research Center Gainesville, Florida, United States
View all 73 locations
View Trial Details
Completed

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

NCT04453085

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
  • Instituto de Genética e Erros Inatos do Metabolismo - IGEIM São Paulo, Brazil
  • Fukuoka Children's Hospital Fukuoka, Japan
View Trial Details

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