Phase 1/2 Clinical Trial of PR006 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)
Status:
Recruiting
Trial end date:
2027-09-01
Target enrollment:
Participant gender:
Summary
Study PRV-FTD101 is a Phase 1/2, multi-center, open-label ascending dose, first-in-human
study that will evaluate the safety and effect of intra-cisternal PR006 administration on
progranulin protein (PGRN) levels in patients with frontotemporal dementia with progranulin
mutations (FTD-GRN). Three escalating dose (low dose, medium dose and high dose) cohorts are
planned. The duration of the study is 5 years. During the first year, patients will be
evaluated for the effect of PR006 on safety, tolerability, immunogenicity, biomarkers, and
efficacy. Patients will follow up for an additional 4 years to monitor safety and changes on
selected biomarkers and clinical outcomes.