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

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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: 26 Sep 2024

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At a glance

  • Drugs Levacetylleucine (Primary)
  • Indications Niemann-Pick disease type C
  • Focus Registrational; Therapeutic Use
  • Sponsors IntraBio

    • Most Recent Events

    • 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
    • 29 Jun 2023 Primary endpoint (Modified Scale for the Assessment and Rating of Ataxia (US only) has been met, According to IntraBio media release.

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