The multicentre, international, open-label, dose-escalation study trial will analyse two dose levels of PBFT02 given as a single injection into the cisterna magna in early symptomatic FTD-GRN patients of the age 35 to 75 years.
Subject enrolment is underway in the trial with two cohorts and based on the findings from these cohorts, an optional third dose level cohort may be enrolled.
Analysing the safety and tolerability of PBFT02 is the primary endpoint while disease biomarkers and clinical outcome measures will comprise the secondary endpoints of the trial.
Furthermore, the two-year trial with a three-year safety extension has obtained CTA acceptance for Clinical Trial Application (CTA) in the US, Brazil and Canada.
PBFT02 uses an adeno-associated virus (AAV) viral vector for delivering a functional GRN gene to patients with gene mutations that encode for PGRN, through ICM administration.
This vector and delivery strategy intends to offer more than usual PGRN protein levels to the CNS for alleviating the progranulin deficiency in GRN gene mutation carriers.
A kind of early onset dementia, FTD has no disease-modifying treatments approved currently.
Passage Bio chief medical officer Mark Forman said: “FTD-GRN is a devastating disease with no approved disease-modifying therapies, and we are hopeful this trial will provide evidence that PBFT02 could become a meaningful treatment option for adults living with FTD-GRN.
“Our approach, which employs the AAV1 vector and ICM administration, provides a potential opportunity to achieve higher than normal levels of PGRN in the CNS, thereby overcoming the PGRN deficiency in GRN mutation carriers with a diagnosis of early symptomatic FTD-GRN.”
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