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

NCT04747431 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2026-05-15

No results posted yet for this 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

DRUG

PBFT02

PBFT02

Sponsors & Collaborators

  • Passage Bio, Inc.

    lead INDUSTRY

Principal Investigators

  • Will Chou, MD · Passage Bio, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
35 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-14
Primary Completion
2028-08-31
Completion
2031-08-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Brazil
  • Canada
  • Portugal

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04747431 on ClinicalTrials.gov