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An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

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Trial Profile

An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 25 Feb 2025

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At a glance

  • Drugs NNZ 2591 (Primary)
  • Indications Telomeric 22q13 Monosomy Syndrome
  • Focus Adverse reactions; Pharmacokinetics
  • Acronyms PMS-001
  • Sponsors Neuren Pharmaceuticals

    • Most Recent Events

    • 22 Feb 2025 According to a Neuren Pharmaceuticals media release, company will present data at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, being held on 18-22 March 2025, in Los Angeles.
    • 27 Nov 2024 Last checked against ClinicalTrials.gov record.
    • 27 Aug 2024 According to a Neuren Pharmaceuticals media release, US Food and Drug Administration granted an End of Phase 2 Meeting for NNZ-2591 to treat Phelan-McDermid syndrome, scheduled for September 2024.

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