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Phase 1/2Active, not recruitingAge-related condition

A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes (upliFT-D)

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 ICM of Adults With FTD and Mutations in the GRN or C9ORF72 Genes

Sponsor
Passage Bio, Inc. (Industry)
Enrolment
30 planned
Conditions
Frontotemporal Dementia; FTD; FTD-GRN; Dementia Frontotemporal; C9orf72
Interventions
PBFT02
Ages
adults, older adults
Registry
NCT04747431

Full record on ClinicalTrials.gov

From the registry

PBFT02 is a gene therapy for frontotemporal dementia intended to deliver a functional copy of the GRN gene to the brain. This study will assess the safety, tolerability and efficacy of this treatment in patients with frontotemporal dementia and mutations in the granulin precursor (GRN) or chromosome 9 open reading frame 72 (C9ORF72) genes

Primary outcomes

  • Number of Participants with Treatment-Related AEs and SAEs
  • Change in Nerve Conduction Velocity and Amplitude from Baseline on Nerve Conduction Studies
  • Change in Cellular and Humoral Response Against the Vector and Transgene in Serum

History

  1. Study completion expected

  2. Primary completion expected

  3. Last registry update

  4. Started

  5. Registered