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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
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Gangliosidoses, GM2 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

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

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
View all 13 locations
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

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