Recruiting studies: Niemann Pick Disease Type C

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 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 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 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.