AviadoBio has opened the multi-centre Phase I/II ASPIRE-FTD trial in the UK, aimed at assessing the gene therapy candidate AVB-101 for individuals with frontotemporal dementia (FTD) harbouring progranulin (GRN) gene mutations (FTD-GRN).
Cambridge University Hospitals National Health Service (NHS) Foundation Trust, focused on FTD care, is actively recruiting participants.
Subject recruitment is also anticipated to commence shortly at University College London. The Advanced Neurotherapies Centre at Cardiff and Vale University Health Board will offer the necessary infrastructure for dosing trial subjects from the UK and other countries.
This open-label trial is aimed at assessing the therapy’s preliminary efficacy and safety.
Leveraging a minimally invasive stereotactic neurosurgical procedure, the gene therapy is said to be delivered only once directly to the brain's thalamus, which connects to areas significantly impacted in FTD-GRN.
Eligible subjects in the trial will undergo this neurosurgical procedure at a centre in the UK, the US, or the European Union (EU) to receive magnetic resonance imaging (MRI)-guided infusions of the therapy into the thalamus.
AviadoBio noted that the ASPIRE-FTD study does not use a placebo. All subjects will receive the AVB-101 treatment, with a subsequent five-year follow-up period.
FTD is said to be a severe, early-onset condition, with symptoms that vary among patients such as cognitive decline, decreased mobility, personality changes, and progressive language loss.
AviadoBio chief medical officer David Cooper said: “Launching ASPIRE-FTD and treating our first patients with AVB-101 have been significant milestones in FTD-GRN research and gene therapy development.
“AviadoBio was built on pioneering research from King’s College London and the UK Dementia Research Institute. The opening of our UK clinical trial sites reflects this strong research heritage, and we’re proud to bring this clinical trial to the UK to make it more accessible to people living with familial FTD in the region.”
In April 2024, the company administered AVB-101 to the first subject in the ASPIRE-FTD trial.
