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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 Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Kaiser Permanente - CA Los Angeles, California, United States
  • UCLA Health Los Angeles, California, United States
  • University of California, San Francisco (UCSF) San Francisco, California, United States
  • University of Colorado Anschutz Aurora, Colorado, United States
View all 33 locations
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Recruiting

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Cedars-Sinai Los Angeles, California, United States
  • Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Duke University Health System Durham, North Carolina, United States
  • Lysosomal & Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
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Recruiting

A Study of Patients With Fabry Disease (US Specific)

NCT06906367

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • UAB Nephrology Research Clinic at Paula Building Birmingham, Alabama, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Emory Genetics Atlanta, Georgia, United States
  • Washington University School of Medicine St Louis, Missouri, United States
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Recruiting

Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting

NCT07109375

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • AUSL Toscana Sud-Est - Ospedale San Donato Arezzo, Italy
  • Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari Bari, Italy
  • Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola Bologna, Italy
  • A.O. Sant'Anna e San Sebastiano Caserta, Italy
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Recruiting

Functional Capacity in Anderson-Fabry Disease Patients

NCT06884358

Conditions
Anderson-Fabry Disease, Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations
  • Spedali Civili Hospital Brescia, Italy
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan, Italy
  • IRCCS Policlinico San Donato San Donato Milanese, Milan, Italy
  • Fondazione IRCCS San Gerardo dei Tintori Monza, Italy
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Recruiting

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Emory Genetics Atlanta, Georgia, United States
  • University of Minnesota Masonic Children's Hospital Minneapolis, Minnesota, United States
  • Atrium Health Levine Children's Hospital Charlotte, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
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Recruiting

Fabry Disease Registry & Pregnancy Sub-registry

NCT00196742

Conditions
Angiokeratoma, Brain Diseases, Brain Diseases, Metabolic, +23 more
Locations
  • University of Alabama Birmingham- Nephrology- Site Number : 840018 Birmingham, Alabama, United States
  • University of Alabama Birmingham- Nephrology- Site Number : 840073 Birmingham, Alabama, United States
  • Phoenix Children's Hospital- Site Number : 840003 Phoenix, Arizona, United States
  • University of Arizona- Site Number : 840015 Tucson, Arizona, United States
View all 284 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
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