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: 25 Apr 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

21 Apr 2025 According to a Zevra Therapeutics Media release, the company has recently sold the Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million to further enhance financial flexibility.
30 Jan 2025 According to a Zevra Therapeutics Media release, data from the study has been accepted for the presentation at the 21st Annual WORLDSymposium™.
28 Nov 2024 Status changed from active, no longer recruiting to completed.

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