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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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6 recruiting trials

for Niemann-Pick Disease, Type C

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 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 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… 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
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