Trial Profile

Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 08 May 2025

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At a glance

30 Apr 2025 Planned End Date changed from 1 Nov 2024 to 1 Dec 2026.
23 Sep 2024 According to IntraBio media release, Based on the results of this study the U.S. Food and Drug Administration approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kilograms.
18 Apr 2024 Results presented at the 76th Annual Meeting of the American Academy of Neurology 2024

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