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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: 25 Apr 2024

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At a glance

  • Drugs L-Acetylleucine (Primary)
  • Indications Niemann-Pick disease type C
  • Focus Registrational; Therapeutic Use
  • Sponsors IntraBio
  • Most Recent Events

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