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A Phase 1b Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacodynamic Effects of a Single Dose of PBFT02 Delivered Into the Cisterna Magna of Adult Subjects With Frontotemporal Dementia and Mutations in the Progranulin Gene

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A Phase 1b Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacodynamic Effects of a Single Dose of PBFT02 Delivered Into the Cisterna Magna of Adult Subjects With Frontotemporal Dementia and Mutations in the Progranulin Gene

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 19 Nov 2024

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At a glance

  • Drugs PBFT 02 (Primary)
  • Indications Frontotemporal dementia
  • Focus Adverse reactions; Pharmacodynamics; Pharmacogenomic; Therapeutic Use
  • Acronyms upliFT-D
  • Sponsors Passage Bio

    • Most Recent Events

    • 13 Nov 2024 According to a Passage Bio media release, 4 patients enrolled in Cohort 2.
    • 24 Oct 2024 According to a Passage Bio media release, the company expects to report the 12 month data for Cohort-1 and interim data for Cohort-2 in 1H 2025.
    • 24 Oct 2024 According to a Passage Bio media release, an oral presentation highlighting the interim safety and biomarker data from this trial was delivered on Thursday 24 Oct 2024, at the European Society of Gene & Cell Therapy (ESGCT) 31st Annual Congress being held October 22-25, 2024, in Rome, Italy.

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