Active not recruitingPHASE2, PHASE3INTERVENTIONAL

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation. Master Protocol DIAN-TU-001

This research study is looking into treatments for a specific type of early-onset Alzheimer's disease that runs in families due to a genetic fault. It aims to see if certain new medications can slow down the progression of memory and thinking difficulties, or improve biological signs of the disease. Researchers will also be checking the safety of these treatments and how well participants tolerate them. The study includes individuals who either know they have this gene fault, or are at risk because it's in their family, and who are near the age when symptoms might start or have very mild Alzheimer's symptoms already. Participants will have regular check-ups, scans, and tests as part of their involvement.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

Washington University School of Medicine

Enrolment target

490

Start

01 Dec 2012

Estimated completion

01 Jul 2028

Alzheimers Disease Dementia Alzheimers Disease, Familial

What is this study about?

This groundbreaking study, called "DIAN-TU-001," focuses on a particular form of Alzheimer's disease that, unlike the more common types, is directly inherited through families because of a specific faulty gene. This type of Alzheimer's usually starts earlier in life. The main goal is to explore whether new medications can slow down the development of memory and thinking problems that are a hallmark of Alzheimer's. Researchers also want to see if these treatments can improve important biological markers linked to the disease, all while carefully monitoring participants' safety and how they respond to the medications.

Participants in this study come from families where this inherited form of Alzheimer's is present. This means they either know they carry the faulty gene, or they are at risk of carrying it because a close family member has it. A unique aspect of this study is that it can include people who haven't yet shown symptoms but are close to the age when symptoms typically begin in their family, or those who have very mild symptoms already. This is possible because, with inherited Alzheimer's, the age when symptoms usually start is quite predictable within affected families.

The insights gained from this study are incredibly important because they could lead to treatments that can be given before or just as symptoms appear, potentially changing the course of this devastating disease. By recruiting participants before or very early in their symptom development, researchers hope to understand if these new medications can intervene effectively at the earliest stages of the disease.

Key takeaways

The study focuses on an inherited form of early-onset Alzheimer's disease.
It tests new medications to see if they can slow disease progression or improve biological markers.
Participants either have, or are at risk for, a specific faulty gene.
It includes people without symptoms, or with very mild symptoms, near their expected age of onset.
Involves regular medical check-ups, brain scans, blood tests, and lumbar punctures.
Participants receive either an investigational drug or a placebo.

Who may be eligible?

To be considered for this study, you would generally need to be between 18 and 80 years old. You must either know you have a gene fault that causes inherited Alzheimer's disease, or be at risk of having it because it runs in your family (meaning a family member has this specific gene problem).

Additionally, your current age needs to fall within a specific window relative to when symptoms are expected to start in your family. This means you could be up to 15 years younger than the expected age of symptom onset, or no more than 10 years older, and might have no symptoms or only very mild memory or thinking problems.

There are also some practical requirements: you should be able to understand and speak a language used in the study, and be capable of having various medical procedures like MRI scans and blood tests. You'll also need a study partner – a friend or family member – who can provide information about your memory and daily abilities.

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 18 and 80 years old?
Does inherited Alzheimer's disease (caused by a gene fault) run in your family?
Are you without symptoms, or do you have only very mild memory changes?
Are you able to undergo MRI brain scans and other medical tests?
Can a friend or family member be your study partner during the research?

Answer every question to see your result.

What does participation involve?

If you join this study, your involvement would include regular visits for assessments. These will involve thorough medical check-ups, cognitive (memory and thinking) tests, and specialised scans such as MRI scans of your brain and PET scans to look at specific disease markers. You would also have blood tests and a lumbar puncture (spinal tap) to collect cerebrospinal fluid, which helps researchers understand changes in the brain.

The study involves taking one of the investigational medications (Gantenerumab, Solanezumab, or E2814, Lecanemab) or a matching placebo (a dummy drug with no active ingredients). These are given to assess their effects. Your health and any potential side effects will be closely monitored throughout the process. The total duration of participation would be ongoing, as the study involves longitudinal assessments, meaning tests and evaluations over an extended period to track changes.

Potential risks and benefits

Participating in research studies like this offers the potential benefit of contributing to our understanding of inherited Alzheimer's disease and possibly helping to develop future treatments. For those receiving the active medications, there's a small chance of personal benefit if the treatment proves effective, though this is not guaranteed due to the experimental nature of the drugs. However, there are potential risks, including side effects from the investigational medications, discomfort from medical procedures like lumbar punctures or MRI scans, and the time commitment required for clinic visits. You will be fully informed about these risks and any potential discomfort before making a decision. Importantly, you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (38)

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
University of California San Diego Medical Center
Verified postcode
La Jolla, United States
USC Keck School of Medicine
Verified postcode
Los Angeles, United States
Yale University School of Medicine
Verified postcode
New Haven, United States
Emory University
Verified postcode
Atlanta, United States
Advocate Lutheran General Hospital
Verified postcode
Park Ridge, United States
Indiana University School of Medicine
Verified postcode
Indianapolis, United States
Washington University in St. Louis
Verified postcode
St Louis, United States
University of Pittsburgh
Verified postcode
Pittsburgh, United States
Butler Hospital
Verified postcode
Providence, United States
Kerwin Medical Center
Verified postcode
Dallas, United States
University of Washington
Verified postcode
Seattle, United States

Common questions

What is 'inherited Alzheimer's disease'?

It's a rare form of Alzheimer's caused by a faulty gene passed down in families, typically starting earlier in life than other types.

What are 'investigational products'?

These are medications currently being tested in research to see if they are safe and effective, and are not yet approved for general use.

What does a 'placebo' mean?

A placebo is a 'dummy' treatment that looks like the actual drug but contains no active ingredients. It helps researchers understand if the drug itself is making a difference.

What is a 'biomarker'?

Biomarkers are measurable signs in the body, like substances in blood or brain scan results, that can indicate the presence or progression of a disease.

Can I still participate if I don't have symptoms?

Yes, the study includes individuals who are at risk for inherited Alzheimer's and are still without symptoms, but are near their expected age of symptom onset.