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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

Recruiting

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Mayo Clinic Rochester, Minnesota, United States
  • The Royal Melbourne Hospital Parkville, Victoria, Australia
  • First Faculty of Medicine, Charles University Hospital Prague Prague, Czechia
  • University of Giessen Giessen, Germany
View all 14 locations
View Trial Details
Recruiting

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

NCT06328608

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Phoenix Children's Phoenix, Arizona, United States
  • Emory Genetics Clinical Trials Center Atlanta, Georgia, United States
  • University of Iowa Iowa City, Iowa, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
View Trial Details
Recruiting

Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT06941025

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
Locations
  • No physical study sites - Decentralized, web-based registry Washington D.C., District of Columbia, United States
  • No physical study sites - Decentralized, web-based registry Berlin, Germany
  • No physical study sites - Decentralized, web-based registry Rome, Italy
  • No physical study sites - Decentralized, web-based registry Madrid, Spain
View Trial Details
Recruiting

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

NCT05710692

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Fukuoka University Chikushi Hospital Chikushino-shi, Fukuoka, Japan
  • Tohoku University Hospital Sendai, Miyagi, Japan
  • University of the Ryukyu Hospital Nishihara, Okinawa, Japan
  • Osaka University Hospital Suita, Osaka, Japan
View Trial Details

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