Ongoing, recruitingHuman Pharmacology (Phase I)- First administration to humansInterventional

Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer’s Disease (AD)

This study, called 'Hero,' is investigating a new medicine called ION269. It's for adults with Down Syndrome who have a higher chance of developing Alzheimer's disease. Since this is the first time humans are being given this medicine, the main goals are to make sure it's safe to use and to understand how the body processes it. This is an early-stage study, meaning researchers are taking careful steps to learn about the new medicine in a small group of people before potentially moving on to larger studies. The hope is to find new ways to help people with Down Syndrome who are at risk of Alzheimer's.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- First administration to humans

Sponsor

Ionis Pharmaceuticals Inc.

Enrolment target

Start

10 Oct 2024

Alzheimer’s Disease in Individuals with Down Syndrome

What is this study about?

This study, known as the 'Hero' trial, is looking at a new potential medicine called ION269. It's designed specifically for adults who have Down Syndrome and are also at risk of developing Alzheimer's disease. Many people with Down Syndrome have a higher chance of getting Alzheimer's as they get older, and researchers are constantly looking for ways to prevent or slow down this condition.

The main purpose of this early-stage study is to discover if ION269 is safe for people to take, and also to understand how the body uses and processes the medicine. This is crucial information when a new medicine is first being tested. Think of it like taking the first careful steps with a new product – you want to make sure it works as expected and doesn't cause harm.

Because this is the very first time the medicine is being given to people, it’s called a 'Phase I' study. These studies are typically small and focus on safety and understanding rather than seeing if the medicine cures a disease. The information gathered from this study will help decide if ION269 can be tested in more people in the future to see if it actually has a positive effect on Alzheimer's disease in those with Down Syndrome.

Key takeaways

This study is testing a new medicine (ION269) for adults with Down Syndrome at risk of Alzheimer's.
It's a very early-stage study, focusing on safety and how the body handles the medicine.
Participants will receive the study medicine and have regular health checks.
Anyone aged 18 or over, with Down Syndrome, and at risk for Alzheimer's might be eligible.
Your regular medical care will not be affected if you join or leave the study.
The goal is to learn if this new medicine could be a safe option for future Alzheimer's research.

Who may be eligible?

This study is for adults aged 18 and older. Both men and women can take part. The most important thing is that participants must have Down Syndrome and be considered at risk for Alzheimer's disease. This 'risk' would be something your doctor would help determine, likely based on certain factors or tests.

There might be other specific health requirements or conditions that would mean someone cannot take part. For example, if someone has certain other health problems or is taking specific medications, they might not be suitable for the study to ensure their safety and the accuracy of the study results. These details would be discussed with you and your doctor if you express interest.

Essentially, the research team needs to make sure that the people joining are healthy enough for the new medicine and that the study results are clear. Your doctor, and the study team, would go through all the detailed requirements to see if this study is a good fit for you or your loved one.

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?
Do you have Down Syndrome?
Has your doctor said you might be at risk for Alzheimer's disease?
Are you able to attend regular appointments at a clinic?
Are you comfortable with taking a new, experimental medicine?
Are you able to understand and agree to the study requirements (or have a legal guardian who can)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the new medicine, ION269. It's likely that you would need to visit the study clinic often for health checks, blood tests, and other assessments. These tests help the doctors monitor your health, check for any possible side effects, and see how the medicine is working in your body. The team would explain exactly how the medicine is given (e.g., as tablets, injection, etc.).

There would be a regular schedule of appointments over a period of time, followed by perhaps some check-ups after you finish taking the medicine. The total length of your participation, and the number of visits, would be clearly explained by the study team. You would always be told what to expect before agreeing to anything.

Potential risks and benefits

Taking part in this study might offer some potential benefits, such as contributing to new medical knowledge that could help people with Down Syndrome at risk of Alzheimer's in the future. You would also receive close medical attention and monitoring from the study doctors. However, as with any new medicine, there are potential risks. The medicine, ION269, might cause side effects that are currently unknown, or it might not have any benefit for you personally. You would always have the right to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Spain

Common questions

What is Alzheimer's disease?

Alzheimer's disease is a condition that affects the brain, causing problems with memory, thinking, and daily activities. It usually worsens over time.

Why are people with Down Syndrome at risk of Alzheimer's?

Many people with Down Syndrome are born with an extra copy of chromosome 21, which includes a gene linked to the buildup of certain proteins in the brain that are associated with Alzheimer's.

What does 'Phase I' study mean?

A Phase I study means this is the very first time a new medicine is given to people. The main goals are to check if it's safe and how the body handles it, rather than to see if it cures a condition.

Will this medicine cure Alzheimer's?

It's too early to say if this medicine will cure or even treat Alzheimer's. This study is focused on safety and understanding how the medicine works in the body. If it looks promising, it might be tested in larger studies later to see if it can help with the disease.

Do I have to pay to join the study?

No, you do not have to pay to take part in a clinical study. All study-related care, tests, and medicines are typically provided free of charge by the study sponsors.