RecruitingPHASE1, PHASE2INTERVENTIONAL

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)

This research is looking into a new gene therapy called AVB-101 for a specific type of dementia called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). This condition affects younger people and leads to problems with a brain protein called progranulin. The study wants to find out if AVB-101 is safe, can restore this protein to normal levels, and potentially slow down the disease. All participants will receive a one-off treatment delivered directly to the brain, and doctors will monitor them for five years. The study involves people aged 30 to 75 who have FTD-GRN and meet specific health requirements.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

AviadoBio Ltd

Enrolment target

Start

30 Aug 2023

Estimated completion

21 Mar 2030

Frontotemporal Dementia FTD FTD-GRN Dementia, Frontotemporal

What is this study about?

This study is looking into a new treatment for a specific type of dementia called Frontotemporal Dementia with Progranulin Mutations, or FTD-GRN. FTD is a brain condition that gets worse over time and can affect a person's behaviour, speech, and movement.

In FTD-GRN, these problems happen because there isn't enough of a vital protein called progranulin in the brain. This lack of protein causes brain cells to die, which then affects how the brain works. The new treatment, called AVB-101, is a type of 'gene therapy'. This means it's designed to help the body make more of the missing progranulin protein.

The main goals of this study are to: 1) check if AVB-101 is safe for people with FTD-GRN, 2) see if it can bring progranulin levels back to normal in the brain, and 3) explore if it can help slow down or even stop the disease from getting worse. This is an early-stage study, meaning it's one of the first times this treatment is being tested in people.

Key takeaways

This study is for people with a specific type of Frontotemporal Dementia called FTD-GRN.
It's testing a new gene therapy, AVB-101, to see if it's safe and can help raise progranulin levels.
Participants will receive a one-time treatment delivered directly into the brain.
The study lasts for five years after the treatment.
There's no placebo; everyone gets the active treatment.
It's an early-stage study, so the full effects and risks are still being learned.

Who may be eligible?

The study is looking for both men and women between the ages of 30 and 75. To be considered, you must have been diagnosed with Frontotemporal Dementia due to a specific genetic change (a GRN mutation).

Doctors will also check how your dementia is affecting you, looking for certain signs related to changes in behaviour or language. You must also be able to follow all the study instructions and attend appointments, and you'll need to have a chosen study partner who can help support you and provide information during the study.

There are also some reasons why you might not be able to join. For example, if your dementia is very severe, or if you have other health conditions that could affect your brain or make the treatment unsafe. You also can't have had certain other experimental treatments or gene therapies before.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 30 and 75 years old?
Have you been diagnosed with Frontotemporal Dementia caused by a GRN gene mutation?
Are you able and willing to attend all study visits and follow instructions?
Do you have a family member or friend who can be your 'study partner'?
Do you have any other serious health conditions that might prevent you from joining?
Have you had any other gene therapy or similar experimental treatments before?

Answer every question to see your result.

What does participation involve?

If you join this study, you won't receive a 'dummy' treatment; everyone will receive the actual study treatment, AVB-101. This treatment is given just once, directly into the brain through a procedure. After this single treatment, doctors will closely monitor your health and how you're responding for a period of five years. This will involve regular check-ups, tests, and assessments to see how you are doing.

Potential risks and benefits

Participating in research studies like this can have potential benefits, such as gaining access to a new, experimental treatment that might help with your condition, and contributing to medical knowledge benefiting others. However, there are also potential risks involved, including those related to the procedure to deliver the treatment, and possible side effects from AVB-101 itself, which are not yet fully known. It's very important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (19)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

The Ohio State University (OSU) Wexner Medical Center
Verified postcode
Columbus, United States· Recruiting
Vanderbilt University Medical Centre
Verified postcode
Nashville, United States· Recruiting
Houston Methodist Hospital
Verified postcode
Houston, United States· Recruiting
UZ Leuven
Verified postcode
Leuven, Belgium· Recruiting
Sunnybrook Health Sciences Centre
Verified postcode
Toronto, Canada· Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta
Verified postcode
Milan, Italy· Recruiting
Amsterdam UMC
Verified postcode
Amsterdam, Netherlands· Recruiting
NEURO-CARE Sp. z o.o. Sp. Komandytowa
Verified postcode
Katowice, Poland· Recruiting
Neurologia Slaska Centrum Medyczne
Verified postcode
Katowice, Poland· Recruiting
Uniwersyteckie Centrum Kliniczne, SUM w Katowicach
Verified postcode
Katowice, Poland· Recruiting
Euromedis Sp. z o.o.
Verified postcode
Szczecin, Poland· Recruiting
Centrum Medyczne NeuroProtect Sp z o.o.
Verified postcode
Warsaw, Poland· Recruiting

Common questions

What is FTD-GRN?

FTD-GRN is a type of Frontotemporal Dementia caused by a change in a specific gene, leading to low levels of a protein called progranulin in the brain.

What is gene therapy?

Gene therapy is a new kind of treatment that aims to correct problems with genes to help the body work correctly, in this case, by hopefully increasing progranulin protein levels.

Is AVB-101 a cure for FTD-GRN?

AVB-101 is an experimental treatment being studied to see if it can help treat FTD-GRN, but it is not yet known if it can cure the condition.

Will I get a placebo in this study?

No, there is no placebo in this study. All participants will receive the active study treatment, AVB-101.

How long will I be involved in the study?

After receiving the one-time treatment, you will be monitored by the study team for five years.

How to find out more

AviadoBio Clinical Trials

clinicaltrials@aviadobio.com +44 203-089-7917 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.