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An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

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Trial Profile

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

Status: Discontinued
Phase of Trial: Phase II/III

Latest Information Update: 30 Sep 2024

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At a glance

  • Drugs Trofinetide (Primary)
  • Indications Rett syndrome
  • Focus Adverse reactions; Pharmacokinetics
  • Acronyms DAFFODIL
  • Sponsors Acadia Pharmaceuticals

    • Most Recent Events

    • 24 Sep 2024 Status changed to discontinued.
    • 18 Apr 2024 Final results assessing the safety tolerability and exploratory efficacy of long-term treatment with trofinetide in girls aged 2-4 years with Rett syndrome presented at the 76th Annual Meeting of the American Academy of Neurology 2024
    • 17 Apr 2024 According to an Acadia Pharmaceuticals media release, data from the study will be presented at the American Academy of Neurology (AAN) 2024 annual meeting, being held this week in Denver, CO.

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