Trial Profile

Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C

Status: Completed

Phase of Trial: Phase II/III

Latest Information Update: 10 Nov 2025

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At a glance

Drugs Arimoclomol (Primary)
Indications Niemann-Pick disease type C
Focus Registrational; Therapeutic Use
Sponsors Orphazyme; Zevra Therapeutics

Most Recent Events

18 Sep 2025 According to a Zevra Therapeutics Media release, data from this study will be presented at International Niemann-Pick Disease Alliance (INPDA) Face-to-Face Meeting Saturday, September 20, 2025
18 Sep 2025 According to a Zevra Therapeutics Media release, data from this study will be presented at the CNS Annual Meeting, Thursday, October 9, 2025; 12:30 to 1:45pm and 5:30 to 7:00pm ET in Charlotte, North Carolina
04 Sep 2025 Results presented in the Zevra Therapeutics Media Release

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