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Clinical trial condition

GM2 Gangliosidosis

Explore clinical trials studying GM2 Gangliosidosis. Study availability and eligibility vary by location and protocol.

10 public trials 4 recruiting studies
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GM2 Gangliosidosis trial results

Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

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
View all 38 locations
View Trial Details
Recruiting

A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

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
View Trial Details
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
View all 23 locations
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Completed

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

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
View Trial Details
Completed

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

NCT03759665

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations
  • University of California - Los Angeles Los Angeles, California, United States
  • Mayo Clinic Rochester, Minnesota, United States
  • NYU Langone School of Medicine New York, United States
  • University of Giessen Giessen, Germany
View Trial Details
Completed

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

NCT04470713

Conditions
AB Variant Gangliosidosis GM2, Brain Diseases, Brain Diseases, Metabolic, +24 more
Locations
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Mayo Clinic - Rochester Rochester, Minnesota, United States
  • Lysosomal and Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • UCL Cliniques Universitaires Saint-Luc Brussels, Belgium
View all 17 locations
View Trial Details

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