NCT00029965
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Atrophy, +29 more
- Locations
-
- National Institutes of Health Clinical Center Bethesda, Maryland, United States
Clinical trial condition
Gangliosidoses, GM2
Explore clinical trials studying Gangliosidoses, GM2. Study availability and eligibility vary by location and protocol.
19 public trials
5 recruiting studies
Gangliosidoses, GM2 trial results
NCT07054515
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 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 Childrens Center - PIN Rochester, Minnesota, United States
- Children's Medical Center Dallas Dallas, Texas, United States
NCT07399704
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
- Locations
-
- Associação Hospitalar de Prot à Infância Dr. Raul Carneiro Água Verde, Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
- Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
NCT00668187
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
- Locations
-
- University of Minnesota - Pediatric Genetics and Metabolism Minneapolis, Minnesota, United States
Recruiting
The Myelin Disorders Biorepository Project
NCT03047369
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
-
- Children's Hospital of Los Angeles Los Angeles, California, United States
- Children's Hospital of Orange County Orange, California, United States
- Stanford University (Lucile Packard Children's Hospital) Palo Alto, California, United States
- University of California, Davis (UC Davis Health) Sacramento, California, 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
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
NCT05758922
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
- Locations
-
- Hospital Pequeno Principe Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
- Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
NCT02851862
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- Massachusetts General Hospital Boston, Massachusetts, United States