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.

77 public trials 13 recruiting studies
Refine in advanced search Browse all conditions

Lysosomal Storage Diseases trial results

Active, Not Recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475

Conditions
Beta-Galactosidase-1 (GLB1) Deficiency, Brain Diseases, Brain Diseases, Metabolic, +23 more
Locations
  • Benioff Children's Hospital Oakland, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Children's Hospital at St. Peter's University Hospital New Brunswick, New Jersey, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
View Trial Details
Active, Not Recruiting

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

NCT05739643

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Children's Hospital of Orange County (CHOC) Orange, California, United States
  • University of Michigan Hospitals - Michigan Medicine Ann Arbor, Michigan, United States
  • Duke University Medical Center Durham, North Carolina, United States
View Trial Details
Active, Not Recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCT03566043

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
Locations
  • University of California San Francisco, Benioff Children's Hospital Oakland, California, United States
  • St. Peter's University Hospital New Brunswick, New Jersey, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders Pittsburgh, Pennsylvania, United States
View Trial Details
Active, Not Recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349

Conditions
Acid Maltase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +17 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • University of California Irvine Health Orange, California, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • University of Kansas Medical Center Research Institute Kansas City, Kansas, United States
View all 29 locations
View Trial Details
Active, Not Recruiting

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

NCT04519749

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of California at San Diego La Jolla, California, United States
  • Emory University Atlanta, Georgia, United States
  • Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania, United States
  • Lysosomal & Rare Disorders Research & Treatment Center, Inc Fairfax, Virginia, United States
View Trial Details

Related conditions