Study to Test Safety and Effects of AVB-101 Gene Therapy for Frontotemporal Dementia Caused by GRN Mutations in Adults Aged 30 to 75

What this Trial Is About

Researchers are studying an investigational gene therapy called AVB-101 for people with Frontotemporal Dementia caused by Progranulin gene mutations FTD-GRN. This form of dementia affects behavior, language, and movement due to low levels of the progranulin protein in the brain, leading to nerve cell death. The study aims to find out if a single treatment with AVB-101 is safe, can restore progranulin levels, and may slow or stop disease progression. Participants will receive a one-time infusion of AVB-101 directly into the brain using MRI-guided stereotaxic surgery. The study includes four groups receiving ascending doses of the gene therapy. There is no placebo group everyone receives the active treatment. After the infusion, participants will be followed for up to 5 years to monitor safety and effects. During the study, participants will undergo regular assessments including cognitive tests like the Mini-Mental State Examination, brain imaging, and blood and cerebrospinal fluid tests to measure progranulin and other markers. Researchers will track any side effects, changes in brain structure, and clinical symptoms over the 5-year period. Participants will also have support from a study partner to help with assessments and study procedures throughout the trial.

A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)