At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Intrathalamic AAV.PGRN administration (procedure), Intrathalamic AVB-101 (genetic)
- How long the study runs
- Study runs about 79 months (dates as stated)
- About the drug or intervention
- Intrathalamic AAV.PGRN administration — procedure: One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain · Intrathalamic AVB-101 — genetic: AVB-101 is made from an adeno-associated virus, serotype 9 (AAV9).
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a gene therapy called AVB-101 in people with a genetic type of frontotemporal dementia caused by changes in the GRN gene (known as FTD-GRN). The researchers want to find out how safe the treatment is and how well people cope with it. It is run by AviadoBio Ltd.
Who can take part
- Adults aged 30 to 75, male or female.
- People who carry a GRN gene change (mutation) known to cause illness.
- Have frontotemporal dementia at a mild or moderate stage, based on study memory and thinking scores.
- Have at least one sign of possible behavioural-variant frontotemporal dementia or primary progressive aphasia (a type affecting language).
- Able and willing to follow the study plan and attend study visits.
- Able to give written agreement (consent) to take part, or have a legal representative who can agree on their behalf where local rules allow. Participants must also agree to name a legal representative in case they lose the ability to consent later.
- Have a study partner (such as a family member or friend) who knows them well, is informed about the study, and can support them and give information about them during the study.
Who may not be able to
- Severe dementia, or other symptoms that would make taking part unsafe or too difficult.
- Any other condition that could cause memory or thinking problems, such as another type of dementia, neurosyphilis, hydrocephalus (fluid on the brain), stroke, small vessel disease in the brain, an underactive thyroid that is not controlled, or vitamin B12 deficiency.
- Something seen on an MRI brain scan at screening that would make the planned surgery unsafe.
- A pattern of brain shrinking seen on the MRI scan that would get in the way of the planned brain surgery route.
- Previous treatment with any gene therapy or cell therapy.
- Taking an experimental medicine within the last 60 days, or within 5 half-lives of that medicine (whichever is longer).
- Any other illness, unusual test result, or treatment that, in the researchers' view, could make taking part unsafe or get in the way of the study.
What taking part involves
- • AVB-101, a gene therapy product, given by neurosurgery (an operation) into the brain, under anaesthetic.
- • Not stated — ask the trial team about how many doses are given or other treatments involved.
Time commitment: Not stated — ask the trial team about how many visits there are, how long the study lasts, and exactly what taking part involves; participants do need to attend scheduled study visits, have MRI scans, and undergo brain surgery under anaesthetic.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 30 Years to 75 Years
- Who
- All
- Number of participants
- 18
- Started
- 2023-08-30
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for frontotemporal dementia
- • Phase1/Phase2 - 18 participants
- • 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)
Who can take part?
- • Ages 30 Years to 75 Years
- • Diagnosed with frontotemporal dementia
Where?
- • Cambridge - Cambridge University Hospitals NHS Foundation Trust
- • Cardiff - University Hospital of Wales
- • London - University College London Hospitals
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 30 Years - 75 Years
- Who can join: All genders
What the study is looking for
- ✓Male or female, 30 to 75 years of age
- ✓Carriers of a pathogenic GRN mutation
- ✓FTD as evidenced by CDR + NACC FTLD global score of 0.5, 1.0, or 2.0
- ✓Presence of 1 or more of the criteria for diagnosis of possible bvFTD or PPA
- ✓Able and willing to comply with all procedures and the study visit schedule
Who cannot take part
- ✗Severe dementia, defined as CDR + NACC FTLD global score of 3.0, or other symptoms that preclude the ability to...
- ✗Clinically significant abnormality on MRI at Screening considered to be a safety concern to Intrathalamic infusion
- ✗Surgically significant pattern of brain atrophy on MRI at Screening that interferes with planned neurosurgical...
- ✗Previous treatment with any gene or cell therapy
- ✗Previous treatment with any investigational medicinal product (IMP) within 60 days or 5 half-lives (whichever is...
See the full criteria
Where Is This Study? (3 UK sites)
Cambridge University Hospitals NHS Foundation Trust
Cambridge CB2 0QQ, United Kingdom
University Hospital of Wales
Cardiff CF14 4XW, United Kingdom
University College London Hospitals
London, United Kingdom
How to Get in Touch
AviadoBio Clinical Trials
Sponsor contactCONTACT