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Niemann-Pick Diseases clinical trials

View clinical trials related to Niemann-Pick Diseases.

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NCT ID: NCT04189601 Withdrawn - Fabry Disease Clinical Trials

Complement Activation in the Lysosomal Storage Disorders

CATALYST
Start date: September 30, 2020
Phase:
Study type: Observational

The lysosomal storage disorders (LSDs) are monogenic disorders associated with inflammation affecting multiple organs, and early death. Few treatments are available that can modify the disease course, and there is an urgent need to identify new steps in pathogenesis that can be targeted therapeutically. The complement system is novel and highly plausible as a primary driver of inflammation and cellular injury in the LSDs. This study assesses the complement activation state in patients with Fabry disease (FD), Gaucher disease (GD) and Niemann-Pick disease, type C (NPC), with comparison to healthy controls. This has the potential for immense clinical benefit through targeted complement inhibition across the full spectrum of lysosomal storage disorders, in which key pathophysiological processes including the inflammatory response to lysosomally 'stored' materials are shared.

NCT ID: NCT03687476 Withdrawn - Clinical trials for Niemann-Pick Disease, Type C

Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease

Start date: May 2020
Phase: Phase 2
Study type: Interventional

This is a Phase-2, multicenter, multiple dose, open-label, 2-part evaluation study which will primarily assess the safety and tolerability of VTS-270 (2-hydroxypropyl beta-cyclodextrin [HP-β-CD]) in pediatric participants with age <4 years.

NCT ID: NCT01306604 Withdrawn - Clinical trials for Niemann-Pick Disease, Type C

Biomarker for Niemann Pick Type C Disease (BioNPC)

BioNPC
Start date: August 20, 2018
Phase:
Study type: Observational

Development of a new MS-based biomarker for the early and sensitive diagnosis of Niemann Pick Type C disease from Blood (plasma)