Recent Updates
Recently added Catalysts

PBFT02

Phase 1

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

Target and mechanism

Molecular targetprogranulin
Target classProtein
ModalitySmall molecule

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment30

FDA Designations

No designations recorded

Clinical trial landscape

PBFT02 · 1 trial · 5 indications

Phase 1 1
NCT04747431A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 GenesFrontotemporal Dementia
ACTIVE NOT_RECRUITING30 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
Frontotemporal Dementia
NCT0474743130
Unlock trial analytics

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)
Unlock Study Endpoints

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
Unlock Study Design Details

Eligibility Criteria

Age Range35 Years to 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
Unlock Eligibility Criteria

Frequently asked questions about PBFT02

What is PBFT02 used for?

PBFT02 is an investigational therapy being developed for frontotemporal dementia (FTD), specifically in participants with mutations in the granulin precursor (GRN) or C9ORF72 genes. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.

What does PBFT02 target?

PBFT02 targets progranulin, a protein involved in cellular processes. By targeting progranulin, PBFT02 aims to address the underlying pathology associated with frontotemporal dementia in patients with GRN or C9ORF72 gene mutations.

Who is developing PBFT02?

PBFT02 is being developed by Passage Bio, Inc., a biopharmaceutical company. The company is conducting a Phase 1 clinical trial to evaluate the safety and efficacy of PBFT02 in patients with frontotemporal dementia.

What phase is PBFT02 in?

PBFT02 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is active but not recruiting participants.

What clinical trials is PBFT02 in?

PBFT02 is being studied in a Phase 1 clinical trial with the identifier NCT04747431. This trial is evaluating PBFT02 in participants with frontotemporal dementia and mutations in the GRN or C9ORF72 genes. The study is active but not recruiting and has an enrollment of 30 participants.

Is PBFT02 the same as other treatments for frontotemporal dementia?

PBFT02 is a distinct investigational therapy targeting progranulin for frontotemporal dementia. It is not the same as other treatments, as it specifically addresses genetic mutations in the GRN or C9ORF72 genes. Its development is separate from other potential therapies for this condition.