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Clinical trial condition

Niemann-Pick Disease, Type C

Explore clinical trials studying Niemann-Pick Disease, Type C. Study availability and eligibility vary by location and protocol.

12 public trials 3 recruiting studies
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Niemann-Pick Disease, Type C trial results

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
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

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

NCT03759639

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Mayo Clinic Rochester, Minnesota, United States
  • University of Giessen Giessen, Germany
  • Ludwig Maximilian University of Munich München, Germany
  • Comenius University in Bratislva Bratislava, Slovakia
View Trial Details
Completed

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT02534844

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +34 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Children's Hospital of Orange County Orange, California, United States
  • University of California San Francisco San Francisco, California, United States
  • University of Colorado Denver Aurora, Colorado, United States
View all 29 locations
View Trial Details
Completed

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

NCT02435030

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University Hospital Copenhagen (Rigshospitalet) Copenhagen, Denmark
  • CHU de Montpellier Montpellier, France
  • Hôpital Trousseau Paris, France
  • Villa Metabolica Mainz Mainz, Germany
View all 16 locations
View Trial Details

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