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Clinical trial condition

Lysosomal Storage Diseases, Nervous System

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

512 public trials 105 recruiting studies
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Lysosomal Storage Diseases, Nervous System trial results

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
Active, Not Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

NCT05206773

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 Birmingham, Alabama, United States
  • UCLA Medical Center_Investigational Site Number: 8400006 Los Angeles, California, United States
  • University of California Irvine Medical Center- Site Number : 8400019 Orange, California, United States
  • Advent Health Orlando_Investigational Site Number: 8400008 Orlando, Florida, United States
View all 58 locations
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Active, Not Recruiting

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Lysosomal Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Hospital de Clinicas de Porto Alegre (HCPA) Porto Alegre, Brazil
  • Hospital Quironsalud Zaragoza Zaragoza, Spain
  • Salford Royal Hospital Salford, United Kingdom
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Active, Not Recruiting

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT05164055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Investigational Site Number : 2500004 Bordeaux, France
  • Investigational Site Number : 2500005 Brest, France
  • Investigational Site Number : 2500008 Clermont-Ferrand, France
  • Investigational Site Number : 2500009 Lille, France
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Active, Not Recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • Wolfson Children's Hospital Jacksonville, Florida, United States
  • Woodruff Memorial Research Building Atlanta, Georgia, United States
  • St. Louis Children's Hospital St Louis, Missouri, United States
View all 17 locations
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