drugset / Trial / NCT04747431
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
Non-randomizedSequentialOpen-labelTreatment
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
Timeline
- Start
- 2021-09-14
- Primary completion
- 2028-08
- Completion
- 2031-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | PBFT02 | Gene therapy (AAV / viral vector) | — | Other |