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

Study Summary

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

Conditions

Frontotemporal Dementia FTD FTD-GRN Dementia Frontotemporal C9orf72

Interventions / Drugs

PBFT02

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment30 participants

Primary Completion—

Lead SponsorPassage Bio, Inc.

Data Retrieved2026-04-11 04:24:05.817340+00:00

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