Clinical Trials Directory

Trials / Conditions / Niemann-Pick Disease, Type C

Niemann-Pick Disease, Type C

21 registered clinical trials studyying Niemann-Pick Disease, Type C — 4 currently recruiting.

StatusTrialSponsorPhase
Active Not RecruitingPhase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Pa
NCT05758922
Azafaros A.G.Phase 2
RecruitingEstablishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
NCT05588167
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)—
RecruitingA Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
NCT05163288
IntraBio IncPhase 3
Enrolling By InvitationScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT05368038
Albert Einstein College of Medicine—
WithdrawnComplement Activation in the Lysosomal Storage Disorders
NCT04189601
Melbourne Health—
WithdrawnSafety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
NCT03687476
Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyPhase 2
CompletedSafety and Efficacy of Miglustat in Chinese NPC Patients
NCT03910621
ActelionPhase 4
TerminatedOpen-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
NCT03879655
Mandos LLCPhase 2 / Phase 3
CompletedN-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
NCT03759639
IntraBio IncPhase 2
CompletedStudy of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
NCT03471143
Washington University School of MedicinePhase 1 / Phase 2
WithdrawnBiomarker for Niemann Pick Type C Disease (BioNPC)
NCT01306604
CENTOGENE GmbH Rostock—
CompletedArimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
NCT02612129
ZevraDenmarkPhase 2 / Phase 3
TerminatedAdrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
NCT04958642
Mandos LLCPhase 2 / Phase 3
CompletedVTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
NCT02534844
Mandos LLCPhase 2 / Phase 3
CompletedA Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
NCT02435030
ZevraDenmark—
CompletedLongitudinal Study of Cognition With Niemann-Pick Disease, Type C
NCT01899950
Mayo Clinic—
CompletedBiomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
NCT00975689
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Phase 1 / Phase 2
TerminatedHematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
NCT00668564
Masonic Cancer Center, University of MinnesotaPhase 2
CompletedApplication of Miglustat in Patients With Niemann-Pick Type C
NCT01760564
National Taiwan University HospitalPhase 3
RecruitingEvaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
NCT00344331
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)—
AvailableEarly Access Program With Arimoclomol in US Patients With NPC
NCT04316637
ZevraDenmark—