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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

Active, Not Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07082543

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
  • Mayo Clinic Children's Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 26 locations
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Active, Not Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT06150820

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Emory Clinic Atlanta, Georgia, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • University of Michigan Ann Arbor, Michigan, United States
  • Children's Hospitals and Clinics of Minnesota Minneapolis, Minnesota, United States
View all 53 locations
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Active, Not Recruiting

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

NCT04127578

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations
  • Mayo Clinic, 13400 E. Shea Boulevard Scottsdale, Arizona, United States
  • Esperanza Clinical, 25220 Hancock Avenue Murrieta, California, United States
  • Rocky Mountain Clinical Research - CenExel - PPDS, 701 East Hampden Avenue Suite 510 Englewood, Colorado, United States
  • K2 Medical Research, 101 Southhall Lane, Suite 150 Maitland, Florida, United States
View all 15 locations
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Active, Not Recruiting

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

NCT05222906

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Yale University School of Medicine - Investigational Site Number: 8400003 New Haven, Connecticut, United States
  • University of Iowa - Investigational Site Number: 8400002 Iowa City, Iowa, United States
  • Texas Oncology - Medical City Dallas Site Number : 8400008 Dallas, Texas, United States
  • Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001 Fairfax, Virginia, United States
View all 22 locations
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Active, Not Recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

NCT06181136

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Iowa Stead Family Children's Hospital Iowa City, Iowa, United States
  • University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
  • Baylor College of Medicine and Texas Children's Hospital Houston, Texas, United States
View Trial Details

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