Dementia Support Line
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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.
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What is the aim of the study?
This study is testing a new medicine called ALN 5288 for people with Alzheimer’s disease. ALN 5288 is a treatment that helps the body make less of a protein called tau. Too much tau in the brain can cause it to clump together, which may lead to memory and thinking problems.
The medicine works by finding and breaking down the instructions inside cells that tell the body to make tau. By reducing tau production, the aim is to lower tau levels, and so slow down the changes in the brain caused by Alzheimer’s disease. The medicine is given as an injection into the space around the spine so it can reach the brain more directly.
What the study wants to find out
The study has two main goals:
- To check if ALN 5288 is safe for people with Alzheimer’s disease and to see how well people can tolerate it.
- To learn how the medicine works inside the body, including what it does and how the body uses and clears it after it is given.
How the study is carried out
This study is a double-blind placebo-controlled trial. This means:
- Some people will get ALN 5288.
- Some people will get a placebo (a dummy treatment that has no medicine in it).
Neither the participant, their study doctor, nor the study team will know who is getting which treatment during the first part of the study. This helps make the results fair.
In the second part of the study, everyone will get ALN 5288, no matter what they received at the start. This part is called an open label extension, because everyone and their doctors will know they are getting the real medicine.
Find more information about why studies use blinding, randomisation and placebo controls.
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What is the aim of the study?
The purpose of this study is to find out whether treatment with VHB937 is safe and beneficial in people with early Alzheimer's disease. The study will evaluate the safety of VHB937, as well as its effects on memory and other thinking abilities, on daily activities, and on changes in the brain. The study will also observe and measure how VHB937 is processed by the body and how the body responds to it.
The aim of this study is to compare the study medicine VHB937 with a placebo (a treatment with no active medicine) to see:
- How VHB937 affects memory, other thinking abilities, daily activities and changes in the brain in people with Early Alzheimer’s Disease
- How safe VHB937 is in people who have early signs of Alzheimer’s disease.
How the study is carried out
This study is a double-blind placebo-controlled trial. This means participants will be selected at random to receive one of the following options:
- An intravenous infusion of VHB937 (low dose)
- An intravenous infusion of VHB937 (high dose)
- An intravenous infusion of Placebo (a 'dummy' treatment, which is an inactive substance with no active therapeutic effect that looks the same as the study drug).
Neither the participant, their study doctor, nor the study team will know who is getting the investigational treatment or placebo. This helps make the results fair.
After the double-blind part of the study participants will be offered the chance to join an extension phase, where they can continue receiving VHB937 in an open label format (meaning everyone receives the real drug, with no placebo). Your study doctor would discuss this with you.
Find more information about why studies use blinding, randomisation and placebo controls.
How is the drug given?
VHB937 or placebo is given by intravenous (IV) infusion, which is a drip into a vein in the arm.
Is the drug already approved for other illnesses?
No, VHB937 is not currently used or sold for any medical condition.
How long does treatment last?
The double-blind part of the study is 72 weeks long (about 1.5 years), followed by an optional open label extension.
What will participants need to do?
During the study, participants will:
- Take tests which check:
- Memory
- Thinking skills (cognition)
- Function
- Daily activities
- Behaviour
- Have positron emission tomography (PET) scans, which take pictures of the brain to check for changes linked to Alzheimer’s disease.
- Have lab tests and checkups which will include:
- blood tests and other lab tests
- neurological exams which help doctors look for any new side effects, such as symptoms or health changes that might happen after receiving the study drug.