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Clinical trial condition

Sphingolipidoses

Explore clinical trials studying Sphingolipidoses. Study availability and eligibility vary by location and protocol.

394 public trials 77 recruiting studies
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Sphingolipidoses 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 27 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

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

NCT03737214

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama at Birmingham - Nephrology Research Clinic Birmingham, Alabama, United States
  • University of California Irvine Irvine, California, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Florida Clinical and Translational Science Institute, UF Clinical Research Center Gainesville, Florida, United States
View all 42 locations
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Active, not recruiting

Long-Term Follow-up of Subjects Who Were Treated With ST-920

NCT05039866

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • University of California, Irvine Irvine, California, United States
  • University of South Florida Tampa, Florida, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • University of Iowa Hospital and Clinics Iowa City, Iowa, United States
View all 13 locations
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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 Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Duke University Health System Durham, North Carolina, United States
  • Lysosomal & Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Westmead Hospital-Cnr Hawkesbury and Darcy Rds Westmead, New South Wales, Australia
  • SphinCS Clinical Science for LSD Höchheim, Germany
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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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