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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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Trial Profile

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: 20 May 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

    • 14 May 2024 According to a Passage Bio media release, the company completed dosing of Cohort 1, with five patients now having received Dose1 of PBFT02
    • 04 Mar 2024 According to a Passage Bio media release, in the first half of 2024, the company expects to complete dosing for Cohort 1 patients; Following the positive data, multiple GRN+ patients have been identified and are being evaluated for eligibility across seven clinical trial sites in Brazil, Canada, the United States and Europe.
    • 20 Dec 2023 According to a Passage Bio media release, management to host a webcast presentation to review interim FTD data today at 8:30 a.m. ET
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