Skip to main content
OpenTrials

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
Refine in advanced search Browse all conditions

Lysosomal Storage Diseases trial results

Active, Not Recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

NCT04573023

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View all 25 locations
View Trial Details
Active, Not Recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

NCT06052800

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Investigational Site Number : 1580004 Taichung, Taiwan
  • Investigational Site Number : 1580005 Tainan, Taiwan
  • Investigational Site Number : 1580001 Taipei, Taiwan
  • Investigational Site Number : 1580003 Taipei, Taiwan
View Trial Details
Active, Not Recruiting

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

NCT06095713

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Fabry disease center Berlin - Charité - Universitätsmedizin Berlin Berlin, Germany
  • Fabry disease center Cologne, Universitätsklinikum Köln Cologne, Germany
  • Fabry disease center Hamburg, Universitätsklinikum Hamburg Hamburg, Germany
  • Fabry disease center Hannover, Universitätsklinikum Hannover Hanover, Germany
View Trial Details
Active, Not Recruiting

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Stanford Hospital- Site Number : 8400006 Stanford, California, United States
  • Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 Minneapolis, Minnesota, United States
  • Advanced Medical Genetics- Site Number : 8400002 Hawthorne, New York, United States
  • Duke University Medical Center- Site Number : 8400004 Durham, North Carolina, United States
View all 16 locations
View Trial Details
Active, Not Recruiting

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

NCT04411654

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • UCSF Benioff Children's Hospital, 747 52nd St Oakland, California, United States
  • University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue Minneapolis, Minnesota, United States
  • Children's Hospital of Pittsburgh, 4401 Penn Avenue Pittsburgh, Pennsylvania, United States
  • Lysosomal & Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
View Trial Details

Related conditions