Recruiting studies: Niemann Pick Type C Disease

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT00344331 UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863 A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pi
Phase 2 · 3 location(s) · Azafaros B.V. · NCT07399704 Retinal Hyperspectral Imaging in Neurodegenerative Diseases
NA · 1 location(s) · Center for Eye Research Australia · NCT07545473 A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years
Phase N/A · 5 location(s) · Sanofi · NCT06192576 Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Phase N/A · 1 location(s) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta · NCT04880356 Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200 GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal G
Phase N/A · 23 location(s) · Fundación Española de Hematología y Hemoterapía · NCT05992532 A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem
Phase 3 · 38 location(s) · Azafaros B.V. · NCT07054515 A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem
Phase 3 · 35 location(s) · Azafaros B.V. · NCT07082725 Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT05588167 A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
Phase 3 · 14 location(s) · IntraBio Inc · NCT05163288

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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.