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PBFT02

Phase 1

Frontotemporal Dementia | Small molecule | Neurology |Passage Bio, Inc.|Last Updated: May 15, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04747431A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 GenesPHASE1 ACTIVE NOT_RECRUITING 30 — — Sep 14, 2021Aug 1, 2031May 15, 202610 United States, Australia +3
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Study Endpoints
Primary Endpoints
Number of Participants with Treatment-Related AEs and SAEs
Up 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 Studies
From 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 Serum
From baseline to 5 years (multiple visits)

Assess ELISpot and antibody titers against AAV1 and against human progranulin

Secondary Endpoints
Change from baseline in CSF and plasma PGRN levels
From baseline to 5 years (multiple visits)
Change from baseline in plasma and CSF neurofilament light chain (NfL) levels
From baseline to 5 years (multiple visits)
Change in Brain anatomy as assessed by MRI
From baseline to 5 years (multiple visits)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTALDrug: PBFT02 Dose 1; Single dose of PBFT02, via intra cisterna magna \*GC/g: gene copy per gram of estimated brain weight
Cohort 2, 3, 4 and 5EXPERIMENTALDrug: PBFT02 Dose 1 or 2; Single dose of PBFT02, via intra cisterna magna \*GC/g: gene copy per gram of estimated brain weight
Interventions
NameTypeDescription
PBFT02DRUGPBFT02
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Eligibility Criteria
Age Range35 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: 1. Documented to be a pathogenic carrier of GRN or C9orf72 mutation 2. Clinical diagnosis of frontotemporal dementia 3. Have a reliable informant / caregiver (and back-up informant / caregiver) who personally speaks with or sees the subject at least weekly 4. Living in the commu...

Countries:United StatesAustraliaBrazilCanadaPortugal
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT04747431Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 24, 2026NCT04747431studyFirstPostDate: changed