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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 (NPC)

NCT07082725

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 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
View all 34 locations
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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
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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

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Mayo Clinic Rochester, Minnesota, United States
  • The Royal Melbourne Hospital Parkville, Victoria, Australia
  • First Faculty of Medicine, Charles University Hospital Prague Prague, Czechia
  • University of Giessen Giessen, Germany
View all 14 locations
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Recruiting

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

NCT05992532

Conditions
Acid Sphingomyelinase Deficiency, Blood Protein Disorders, Brain Diseases, +38 more
Locations
  • Hospital Universitario de Álava Vitoria-Gasteiz, Alava, Spain
  • Hospital Universitario Torrecárdenas Almería, Spain
  • Hospital Son Espases Palma de Mallorca, Balearic Islands, Spain
  • Hospital Parc Taulí Sabadell, Barcelona, Spain
View all 23 locations
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Recruiting

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

NCT06192576

Conditions
Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +19 more
Locations
  • Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002 Chicago, Illinois, United States
  • Children's Hospital Medical Center- Site Number: 001003 Cincinnati, Ohio, United States
  • Nationwide Children's Hospital - PIN- Site Number : 1001-1 Columbus, Ohio, United States
  • Pulse InfoFrame US Inc.- Site Number: 001001 Philadelphia, Pennsylvania, United States
View Trial Details
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
View Trial Details
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

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

NCT05641103

Conditions
ASMD, Acid Sphingomyelinase Deficiency, Brain Diseases, +25 more
Locations
  • Hospital General Universitario de Alicante Alicante, Spain
  • Hospital de Poniente El Ejido, Almería, Spain
  • Hospital Universitario de Badajoz Badajoz, Spain
  • Hospital Vall d'Hebron Barcelona, Spain
View all 14 locations
View Trial Details
Completed

A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

NCT04845958

Conditions
Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +25 more
Locations
  • Investigational Site Number 2500036 Amiens, France
  • Investigational Site Number 2500014 Angers, France
  • Investigational Site Number 2500034 Argenteuil, France
  • Investigational Site Number 2500039 Bayonne, France
View all 40 locations
View Trial Details