NCT06064890

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)

A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: AviadoBio Ltd · Industry
• Sex: All
• Age: 30 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). FTD-GRN is an early-onset form of dementia, a progressive brain disorder that affects behavior, language and movement. These symptoms result from below normal levels of a protein called progranulin (PGRN) in the brain, which leads to the death of nerve cells (neurons), affecting the brain's ability to function. The main questions that the study aims to answer are: 1. Is a one-time treatment with AVB-101 safe for patients with FTD-GRN? 2. Does a one-time treatment with AVB-101 restore PGRN levels to at least normal levels? 3. Could AVB-101 work as a treatment to slow down or stop progression of FTD-GRN? In this study there is no placebo (a dummy pill or treatment used for comparison purposes), so all participants will receive a one-time treatment of AVB-101 delivered directly to the brain, with follow-up assessments for 5 years.

Conditions

• Frontotemporal Dementia
• FTD
• FTD-GRN
• Dementia, Frontotemporal

Interventions

Type	Name	Description
PROCEDURE	Intrathalamic AAV.PGRN administration	One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain
GENETIC	Intrathalamic AVB-101	AVB-101 is made from an adeno-associated virus, serotype 9 (AAV9). AAVs are small viruses that are naturally occurring and do not cause illness or infection on their own. AVB-101 has been modified to contain a copy of the correct (non-mutated) GRN gene, plus some other genetic material to enable the GRN gene to function inside neurons (cells within the brain). AVB-101 has also been modified so that it cannot divide and make new copies of itself (known as 'replication'), which means that it cannot cause disease or a large immune response in your body.

Timeline

• Start date: 2023-08-30
• Primary completion: 2026-04-01
• Completion: 2030-03-21
• First posted: 2023-10-03
• Last updated: 2026-04-13

Locations

19 sites across 9 countries: United States, Belgium, Canada, Italy, Netherlands, Poland, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated device study