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Trial Match

Clinical trials test new treatments — sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

Know your stage or your test results (such as EGFR, HER2 or BRCA)? Match trials to your own profile - nothing you type is saved.

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13 recruiting trials

for Niemann-Pick Type C Disease

Retinal Hyperspectral Imaging in Neurodegenerative Diseases NCT07545473NA1 site📍 Melbourne Center for Eye Research Australia · Tests: Hyperspectral camera 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) NCT07082725Phase 334 sites📍 Oakland📍 Minneapolis📍 Rochester Azafaros B.V. · Tests: Nizubaglustat, Placebo GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma NCT05992532n/a23 sites📍 Vitoria-Gasteiz📍 Almería📍 Palma de Mallorca Fundación Española de Hematología y Hemoterapía A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C NCT05163288Phase 314 sites📍 Rochester📍 Parkville📍 Prague IntraBio Inc · Tests: N-Acetyl-L-Leucine, Placebo National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children NCT06985212n/a1 site📍 Paris Wladimir MAUHIN, Dr UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells NCT02254863Phase 11 site📍 Durham Joanne Kurtzberg, MD · Tests: DUOC-01 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) NCT06192576n/a6 sites📍 Chicago📍 Cincinnati📍 Columbus Sanofi · Tests: Olipudase alfa Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases. NCT04880356n/a1 site📍 Milan Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta · Tests: collection of data Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C NCT00344331n/a1 site📍 Bethesda Eunice Kennedy Shriver National Institute of Child Health a… Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C NCT05588167n/a1 site📍 Bethesda Eunice Kennedy Shriver National Institute of Child Health a… Longitudinal Study of Neurodegenerative Disorders NCT03333200n/a1 site📍 Pittsburgh University of Pittsburgh · Tests: Palliative Care, Hematopoetic Stem Cell Transplantation 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 NCT07054515Phase 339 sites📍 Oakland📍 Minneapolis📍 Rochester Azafaros B.V. · Tests: AZ-3102, Placebo Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease NCT06869499NA1 site📍 Paris Wladimir MAUHIN, Dr · Tests: Blood sampling for dosage
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