A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis	Recruiting	Phase 3	2026-04-03		NCT05163288

1 results · Page 1 of 1

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Organizations	Linked trials
IntraBio Inc	3

14 results · Page 1 of 1

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Research sites	City	Country	Linked trials
Amsterdam UMC	Amsterdam	Netherlands	6
Comenius University in Bratislva	Bratislava	Slovakia	1
First Faculty of Medicine, Charles University Hospital Prague	Prague	Czechia	1
Great Ormond Street Hospital	London	United Kingdom	38
Ludwig Maximilian University of Munich	München	Germany	1
Mayo Clinic	Rochester	United States	664
Royal Free London NHS Foundation Trust	London	United Kingdom	15
Royal Manchester Children's Hospital	Manchester	United Kingdom	32
SphinCS - Institute of Clinical Science in Lysosomal Storage Disorders	Höchheim	Germany	1
The Royal Melbourne Hospital	Parkville	Australia	26
University Hospital Bern Inselspital	Bern	Switzerland	3
University Hospital Münster	Münster	Germany	12
University of Giessen	Giessen	Germany	1
University of Hamburg	Hamburg	Germany	2