NCT00418847
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- The Hospital for Sick Children Toronto, Ontario, Canada
Clinical trial condition
Gangliosidoses
Explore clinical trials studying Gangliosidoses. Study availability and eligibility vary by location and protocol.
28 public trials
6 recruiting studies
Gangliosidoses trial results
Completed
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
NCT01102686
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
- Locations
-
- The Hospital for Sick Children Toronto, Ontario, Canada
NCT00672022
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- Children's National Medical Center Washington D.C., District of Columbia, United States
NCT00006057
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
- Locations
-
- Mount Sinai School of Medicine New York, United States
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
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
Active, Not Recruiting
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
NCT06614569
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- UMass Chan Medical School Worcester, Massachusetts, United States
Active, Not Recruiting
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
NCT04798235
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- Queen's University/Kingston Health Sciences Centre Kingston, Ontario, Canada