RecruitingPHASE2, PHASE3INTERVENTIONAL

A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset AD Caused by a Genetic Mutation

This research is investigating new treatments, called 'investigational study drugs', for a rare type of Alzheimer's disease that runs in families due to a specific genetic change. The main goal is to find out if these drugs can prevent or slow down the build-up of harmful proteins (amyloid beta) in the brain, which are linked to Alzheimer's. The study will also check the safety of these drugs. It's looking at people who are either at risk of developing this inherited form of Alzheimer's or are in its very early stages. The hope is to find ways to treat the disease before severe symptoms appear, potentially changing its course.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

Washington University School of Medicine

Enrolment target

280

Start

22 Nov 2024

Estimated completion

30 Aug 2034

Alzheimers Disease Dementia Alzheimers Disease, Familial

What is this study about?

This important study is looking into new ways to treat a specific type of Alzheimer's disease that is passed down through families. This 'familial' type is very rare and is caused by a faulty gene. If someone has this faulty gene, they are almost certain to develop Alzheimer's, often at a younger age than typical Alzheimer's.

The researchers want to test if new medications can stop or slow down the disease process before it causes noticeable problems. They are especially interested in preventing the build-up of a protein called 'amyloid beta' in the brain, which is a key feature of Alzheimer's. They will use special brain scans to see if the drugs have this effect. Later, they will also look at other signs in the body that show if the disease is progressing.

Participants for this study come from families where this inherited form of Alzheimer's is known. This allows doctors to identify people who are at very high risk, even before they show any symptoms. The study is designed to see if treating people early, when they are still healthy but carry the faulty gene, can make a real difference to their future health.

Key takeaways

This study is for a rare, inherited form of Alzheimer's disease.
It aims to test new drugs to prevent or slow down disease progression.
Participants must have a specific genetic mutation or be at high risk.
The study involves regular clinic visits, scans, and other medical tests.
It helps researchers understand how to treat Alzheimer's before symptoms appear.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, participants must be at least 18 years old. A key requirement is that you either carry a specific faulty gene linked to this inherited type of Alzheimer's, or you are at risk because someone in your immediate family has it, and you haven't yet checked your own genetic status.

Another important factor is your age in relation to when symptoms might typically start in your family. You would need to be within a specific age window (between 25 and 11 years before the expected start of memory or thinking problems in your family). Your memory and thinking abilities must also be normal at the time of joining.

Women who could become pregnant will need to take a pregnancy test and agree to use effective contraception during the study. If you meet these main points, the research team will explain everything to you in detail.

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 at least 18 years old?
Do you have a known faulty gene for inherited Alzheimer's, or is there a strong family history and you are at direct risk?
Are your memory and thinking abilities currently normal?
Are you within the specific age range for your family's expected age of Alzheimer's onset?
Are you able to attend regular clinic appointments and undergo various medical tests?
If you could become pregnant, are you willing to use effective contraception during the study?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive either the investigational study drug or a matching dummy drug (placebo) through injections under the skin. The study will involve regular visits to the clinic for various assessments. These will include check-ups by doctors, tests of your memory and thinking, and special brain scans (like MRI and PET scans) to look at your brain in detail. You will also have samples taken, such as cerebrospinal fluid (from your spine), to check for markers related to Alzheimer's.

The study is divided into two main stages, looking at different aspects of the drug's effect. The total duration of your participation, including follow-up, would be fully explained by the study team. You would also have a 'study partner' – someone who knows you well – attending some of your visits to help with information.

Potential risks and benefits

Taking part in a clinical trial offers the chance to contribute to medical research and potentially gain early access to new treatments that might help. However, as with any medication, there could be side effects from the investigational study drugs, which would be explained to you in full. There's also no guarantee that the treatment will work for you. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (35)

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

University of Alabama in Birmingham
Verified postcode
Birmingham, United States· Recruiting
University of California San Diego Medical Center
Verified postcode
La Jolla, United States· Recruiting
Yale University School of Medicine
Verified postcode
New Haven, United States· Recruiting
Emory University
Verified postcode
Atlanta, United States· Recruiting
Advocate Lutheran General Hospital
Verified postcode
Park Ridge, United States· Recruiting
Indiana University School of Medicine
Verified postcode
Indianapolis, United States· Recruiting
Washington University in St. Louis
Verified postcode
St Louis, United States· Recruiting
New York University Medical Center
Verified postcode
New York, United States· Recruiting
University of Pittsburgh
Verified postcode
Pittsburgh, United States· Recruiting
Butler Hospital
Verified postcode
Providence, United States· Recruiting
Kerwin Research and Memory Center
Verified postcode
Dallas, United States· Recruiting
University of Washington
Verified postcode
Seattle, United States· Recruiting

Common questions

What is 'familial Alzheimer's disease'?

It's a very rare type of Alzheimer's that runs in families, caused by a faulty gene passed down from a parent. It often starts earlier in life than typical Alzheimer's.

What is 'amyloid beta' and why is it important?

Amyloid beta is a protein that can build up in the brain, forming sticky plaques. These plaques are thought to be a key cause of damage in Alzheimer's disease.

Will I know if I'm getting the real drug or a dummy pill (placebo)?

No, in this type of study, participants usually don't know whether they are receiving the active drug or a placebo. This helps ensure unbiased results.

What is a 'study partner'?

A study partner is often a family member or close friend who comes with you to some appointments and can help answer questions about your health and daily life.

Can I stop participating in the study at any time?

Yes, you have the right to withdraw from the study at any point, for any reason, without it affecting any future medical care you receive.

How to find out more

Jamie Bartzel

dianexr@wustl.edu 844-DIANEXR (342-6397) Official trial record

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