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Clinical Trial 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 progranulin gene (FTD-GRN).


Clinical Trial Description

PBFT02 is an adeno-associated viral vector serotype 1 carrying GRN, the gene encoding for human progranulin, formulated as a solution for injection into the cisterna magna. This is a global interventional, multicenter, open-label, single-arm, dose-escalation study of PBFT02 delivered as a one-time dose administered into the cisterna magna to patients with FTD-GRN. Subjects aged ≥ 35 and ≤ 75 years with early symptomatic FTD-GRN may be enrolled into the study. Two dose levels of PBFT02 will be studied in patients with FTD-GRN. The study will sequentially enroll 2 cohorts. An optional third dose level cohort may be enrolled based on the results of the first two cohorts. This is a 5-year study, with a 2-year main study, followed by a 3-year safety extension. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04747431
Study type Interventional
Source Passage Bio, Inc.
Contact Patient/Family Inquiries
Phone 267-866-0113
Email patientservices@passagebio.com
Status Recruiting
Phase Phase 1/Phase 2
Start date September 14, 2021
Completion date August 2027

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