Niemann-Pick Disease, Type C Clinical Trials

24 clinical trials for Niemann-Pick Disease, Type C across international registries, including 5 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C — RECRUITING
• Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C — RECRUITING
• ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program — RECRUITING
• A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C — RECRUITING · PHASE3
• Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II study — RECRUITING
• Efficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. — ACTIVE_NOT_RECRUITING · PHASE3
• Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease — COMPLETED · PHASE2
• Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C — COMPLETED · PHASE1
• Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study — TERMINATED · PHASE3
• Safety and Efficacy of Miglustat in Chinese NPC Patients — COMPLETED · PHASE4
• Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C — COMPLETED · PHASE2
• Early Access Program With Arimoclomol in US Patients With NPC — OTHER
• Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 — TERMINATED · PHASE2
• N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) — COMPLETED · PHASE2
• Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease — TERMINATED · PHASE2
• VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease — COMPLETED · PHASE2
• Biomarker for Niemann Pick Type C Disease (BioNPC) — WITHDRAWN
• Complement Activation in the Lysosomal Storage Disorders — WITHDRAWN
• Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease — WITHDRAWN · PHASE2
• Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism — TERMINATED · PHASE2
• A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C — COMPLETED
• Longitudinal Study of Cognition With Niemann-Pick Disease, Type C — COMPLETED
• Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine — COMPLETED · PHASE1
• Application of Miglustat in Patients With Niemann-Pick Type C — COMPLETED · PHASE3

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