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

NCTID	NCT04747431 (View at clinicaltrials.gov)
Description	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 (Show More)
Development Status	Active
Indication	Frontotemporal Dementia, FTD, FTD-GRN, C9orf72
Disease Ontology Term	DOID:0060672
Compound Name	PBFT02
Sponsor	Passage Bio, Inc.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	25
Results Posted	Not Available

Target Gene/Variant	GRN
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracisterna magna
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV1
Editor Type	none
Dose 1	3.3E10 GC/g brain weight
Dose 2	1.1E11 GC/g brain weight
Dose 3	2.2E11 GC/g brain weight
Dose 4
Dose 5

No.of Trial Sites	7
Locations	Canada,United States,Brazil,Portugal

Has US IND	True
FDA Designations
Recent Updates	12-month data from Dose 1 and interim safety and biomarker data from Dose 2 expected in 2H 2025; plan to seek regulatory feedback on FTD-GRN pivotal trial design in 1H 2026