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NCT04747431

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

Active, Not Recruiting
PHASE1Ages 35–75InterventionalTreatment
Passage Bio, Inc.
~30 participants
Updated 2026-05-15 on ClinicalTrials.gov
What's tested:PBFT02

At a glance

Recruiting sites
0 of 10 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants with Treatment-Related AEs and SAEs
Measured over Up to 5 years (multiple visits)
+2 more outcomes measured

Conditions

Frontotemporal Dementia
FTD
FTD-GRN
Dementia Frontotemporal
C9orf72

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Frontotemporal DementiaC9orf72 GeneGRN Gene

Where it's being run

10 sites across 9 states
Brazil2
Michigan1
Pennsylvania1
Tennessee1
Texas1
Victoria1
Ontario1
Quebec1

Study leadership

  • Will Chou, MD · STUDY_DIRECTOR · Passage Bio, Inc.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Exclusion

Platelet count \> 100,000 per uL
INR \< 1.5
aPTT \< 40 seconds 30. Use of anticoagulants in the 2 weeks prior to screening 31. Hypersensitivity or contraindications to corticosteroid use 32. Known or suspected intolerance or hypersensitivity to PBFT02 or any of its ingredients or to closely related compounds 33. Any condition expected to increase risk of thrombosis 34. Hypersensitivity or contraindications to apixaban

What this trial measures

  • Number of Participants with Treatment-Related AEs and SAEsUp to 5 years (multiple visits)

    Assess the number of treatment-related adverse events (AEs) and serious adverse events (SAEs)

  • Change in Nerve Conduction Velocity and Amplitude from Baseline on Nerve Conduction StudiesFrom baseline to 5 years (multiple visits)

    Assess changes in nerve conduction velocity in the distal segments of the sural, radial, and median sensory nerves and peroneal motor nerve as measured on conventional nerve conduction studies.

  • Change in Cellular and Humoral Response Against the Vector and Transgene in SerumFrom baseline to 5 years (multiple visits)

    Assess ELISpot and antibody titers against AAV1 and against human progranulin