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
Gangliosidosis, GM1
Explore clinical trials studying Gangliosidosis, GM1. Study availability and eligibility vary by location and protocol.
15 public trials
4 recruiting studies
Gangliosidosis, GM1 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
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
NCT06539169
- Conditions
- Alpha-Thalassemia, Alzheimer Disease, Amyloidosis, +104 more
- Locations
-
- xCures Los Altos, California, United States
Not yet recruiting
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
NCT07479953
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
Not specified
Enrolling by invitation
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT05368038
- Conditions
- Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
- Locations
-
- Maimonides Medical Center Brooklyn, New York, United States
- NYU Langone Hospital - Brooklyn Brooklyn, New York, United States
- North Shore University Hospital Manhasset, New York, United States
- NYU Langone Health - Tisch Hospital 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
NCT04041102
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
- Montreal Children's Hospital Research Institute - McGill University Montreal, Quebec, Canada
NCT05109793
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland, California, United States
- Mayo Clinic Rochester Rochester, Minnesota, United States
- Hospital Pequeno Principe Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil