Niemann-Pick disease type C
MONDO:0018982NPC is a complex lipid storage disease mainly characterized by the accumulation of unesterified cholesterol in the late endosomal/lysosomal compartment.
Also known as: NPC, Niemann Pick Disease Type C
19 clinical trials for this condition and its sub-types, 12 tagged with Niemann-Pick disease type C itself.
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Browse by category →Sub-types of Niemann-Pick disease type C
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Niemann-Pick disease, type C1 2 trials
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Niemann-Pick disease, type C2 1 trial
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New pill shows promise for rare brain disorders in early trial
Disease control CompletedThis study tested an oral drug called AZ-3102 in 13 people with GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) or Niemann-Pick type C disease. The main goal was to check safety and how the drug moves through the body over 12 weeks. It was a randomized, double-blind, placebo-c…
Phase 2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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Promising drug tested for rare liver disease in newborns
Disease control CompletedThis early-phase study tested a drug called adrabetadex (VTS-270) given through a vein to treat liver disease in infants with Niemann-Pick disease type C, a rare and fatal genetic disorder. Four babies up to 6 months old received multiple doses to see if the drug could lower abno…
Phase 1/2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC