Dementia Support Line
Testing a gene therapy (AVB-101) for a genetic form of Frontotemporal Dementia
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Use the information below to reference this clinical trial when speaking with your healthcare professional or research team.
Note: the information presented on the Clinical Trial Finder is for information only. To join a clinical trial you must meet the eligibility criteria set by the study and the research team will discuss this with you.
This trial is open to recruitment at the following locations. Contact your preferred location at their email address.
Contact the Clinical Trials team
- Cambridge University Hospitals NHS Foundation Trust: [email protected]
- University College London: [email protected]
Trial description
What is the goal of this study?
This study is looking at a new gene therapy called AVB 101. It is being developed to treat people with Frontotemporal Dementia who carry a Progranulin Mutation (FTD GRN).
FTD GRN is a rare type of dementia that usually starts earlier in life. It affects behaviour, language, and movement. People with this condition have very low levels of a protein called progranulin (PGRN) in the brain. Without enough PGRN, brain cells (neurons) begin to die, and the brain cannot work properly.
AVB 101 is designed to help the body make more PGRN, which may help protect brain cells.
What is the study trying to find out?
The study has three main questions:
- Is a one time dose of AVB 101 safe for people with FTD GRN?
- Does AVB 101 increase PGRN levels in the brain back to normal or close to normal?
- Can AVB 101 help slow down or stop the worsening of FTD GRN?
What treatment will participants receive?
This study does not use a placebo. This means everyone who joins the study will receive AVB 101.
AVB 101 is a gene therapy which is given once, directly into the brain, using a special medical procedure. After this one time treatment, participants will have regular check-ups at the study centre for 5 years so doctors can see how they are doing and how well the treatment is working.
Trial recruitment and assessment sites are in London and Cambridge. However, all participants will be required to travel to the trial surgical site in Cardiff to receive AVB-101.
What will participants be asked to do during the study?
During the study, participants will:
- Have some laboratory tests, such as blood tests. These tests help doctors check for any new side effects, such as symptoms or health changes that might happen after receiving the study medicine.
- Have brain scans called MRI scans. These scans take detailed pictures of the brain to help doctors check brain health and safety.
- Complete tests that look at memory, thinking, and emotional wellbeing.
If participants would like more details about the tests, procedures, or number of visits, the study team can provide full information.
It is a one-time treatment of AVB-101 with follow-up assessments for 5 years.
Eligibility criteria
Who can take part
- People who have a diagnosis of frontotemporal dementia and who have a progranulin genetic mutation (confirmed with a genetic test).
- People who have an identified, informed study partner who is able and willing to support the participant in the study and to provide assessments of the participant during the study.
- People who are able and willing to comply with all procedures, attend study visits and give written informed consent.
Who can’t take part
People cannot take part in the study if they have, or have experienced:
- Severe dementia or other symptoms that prevent them from following study procedures and/or pose unacceptable safety risk
- Any disease that may cause cognitive impairment unrelated to progranulin mutations, such as other causes of dementia, neurosyphilis, hydrocephalus, stroke, small vessel ischemic disease, uncontrolled hypothyroidism, or vitamin B12 deficiency
- Previous treatment with any gene or cell therapy
- Previous treatment with any investigational medicinal product within 60 days prior to study drug treatment
Other specific medical criteria will apply, which a study doctor will discuss with you and evaluate.
Clinical trial team
Age range
Recruitment start
Recruitment end
Disease type
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Trial locations
Eligibility criteria
Who can take part
- People who have a diagnosis of frontotemporal dementia and who have a progranulin genetic mutation (confirmed with a genetic test).
- People who have an identified, informed study partner who is able and willing to support the participant in the study and to provide assessments of the participant during the study.
- People who are able and willing to comply with all procedures, attend study visits and give written informed consent.
Who can’t take part
People cannot take part in the study if they have, or have experienced:
- Severe dementia or other symptoms that prevent them from following study procedures and/or pose unacceptable safety risk
- Any disease that may cause cognitive impairment unrelated to progranulin mutations, such as other causes of dementia, neurosyphilis, hydrocephalus, stroke, small vessel ischemic disease, uncontrolled hypothyroidism, or vitamin B12 deficiency
- Previous treatment with any gene or cell therapy
- Previous treatment with any investigational medicinal product within 60 days prior to study drug treatment
Other specific medical criteria will apply, which a study doctor will discuss with you and evaluate.