AuthorisedPhase II and Phase III (Integrated)Interventional

A Phase II/III Multicenter Randomized, Double-Blind, Placebo-Controlled, Two-Stage Adaptive Design, Platform Trial of Investigational Treatments for Primary Prevention of Disease Progression in Dominantly Inherited Alzheimer’s Disease

This research is looking at new treatments for a rare type of Alzheimer's called Dominantly Inherited Alzheimer's Disease (DIAD). This form of Alzheimer's runs in families and can start at a younger age. The study aims to find out if these new medicines can help prevent or delay the disease from getting worse before people even show symptoms. Researchers will be checking for changes in the brain, like unusual protein build-ups, to see how the treatments are working. By understanding these changes, they hope to find ways to protect the brain and keep it healthy for longer.

At a glance

Status

Authorised

Phase

Phase II and Phase III (Integrated)

Sponsor

Washington University School Of Medicine

Enrolment target

Start

30 Jun 2025

Dominantly Inherited Alzheimer’s Disease (DIAD)

What is this study about?

This study is about a specific type of Alzheimer's disease called Dominantly Inherited Alzheimer's Disease, or DIAD. This illness is quite rare and is passed down through families, often leading to symptoms at a younger age than typical Alzheimer's. The main goal of this research is to see if new medicines can help stop or slow down the disease even before a person starts to show any memory problems or other symptoms. Think of it like trying to prevent a cold before you even sneeze.

The researchers are particularly interested in understanding the very early changes that happen in the brains of people with DIAD. They'll be looking for tiny signs, called 'biomarkers', that indicate the disease process is starting. These might include checking for certain unusual proteins or changes in brain scans. By tracking these biomarkers, they hope to learn if the new treatments are effectively tackling the disease at its root, before it has a chance to cause significant damage.

This study is designed in two main parts, or 'stages'. In the first stage, they will focus on whether the treatments can reduce problematic protein build-up in the brain. If a treatment shows promise, it might then move to the second stage, which would look at other brain changes over a longer time. This careful, step-by-step approach helps ensure that the researchers gather the best possible information about how these new medicines work.

Key takeaways

This study is for a specific, inherited type of Alzheimer's disease (DIAD).
It aims to test new medicines to stop or slow the disease before symptoms appear.
Researchers will use brain scans and other tests to look for early changes in the brain.
You might receive an active medicine or a placebo; neither you nor your doctor will know which.
Participation involves regular hospital visits for assessments and treatments.
You can withdraw from the study at any point without affecting your medical care.

Who may be eligible?

To join this study, people generally need to be 18 years old or older. The study is open to both men and women, but it's specifically for those who have Dominantly Inherited Alzheimer's Disease (DIAD), or are at high risk of developing it because it runs in their family.

Because this is a very specific type of Alzheimer's, not everyone with Alzheimer's disease will be able to take part. The research focuses on people who have inherited the faulty gene that causes DIAD. This means if Alzheimer's doesn't run in your family in this specific way, or if you already have noticeable symptoms that are too advanced, this particular study might not be the right fit for you. The research team will do medical tests to make sure participants meet all the requirements for the study.

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 18 years old or older?
Does Dominantly Inherited Alzheimer’s Disease (DIAD) run in your family?
Have you been diagnosed with DIAD, or are you considered at high risk for it?
Are you able to attend regular appointments at a study centre?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be given either an investigational new medicine or a placebo, which looks like the medicine but contains no active drug. This is given to see if the new medicine works better than no treatment at all. You won't know whether you're receiving the actual medicine or the placebo, and neither will your study doctor, until the study is over.

You will need to attend appointments at a study centre for various medical assessments. These may include regular brain scans (like PET scans to check for protein build-up and MRI scans to look at brain structure), blood tests, and possibly spinal fluid tests. These tests help researchers track how the disease is progressing and if the treatment is having an effect. The exact number of visits and tests will be explained to you in detail by the study team, and they will tell you how long your participation in the study is expected to last, which could be several years.

Potential risks and benefits

Taking part in a clinical trial might offer the chance to receive a new treatment that isn't yet available to everyone, which could potentially help slow down or prevent the progression of your condition. It also contributes valuable information that could help others in the future. However, there are potential risks, as with any medical treatment. The investigational medicines might have side effects, which you would discuss with the study team. Some of the tests, like scans or blood draws, also carry their own small risks or discomforts. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

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

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Unverified
Spain
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Unverified
France
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Unverified
Germany
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Unverified
Italy
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Unverified
Netherlands

Common questions

What is Dominantly Inherited Alzheimer’s Disease (DIAD)?

It's a rare form of Alzheimer's disease that runs in families and often starts at a younger age. It's caused by a specific faulty gene passed down from a parent.

Why is this study 'double-blind'?

Double-blind means neither you nor the doctors know if you're getting the active medicine or a placebo. This helps make sure the results are fair and unbiased.

What kind of tests will I have?

You'll likely have brain scans (like PET and MRI), blood tests, and possibly spinal fluid tests to check for changes in your brain and how the treatment is working.

What is a 'biomarker'?

Biomarkers are tiny signs in your body, like certain proteins or changes seen on scans, that can tell doctors about your health or how a disease is developing.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, for any reason, without it affecting your regular medical care.