RecruitingPHASE1INTERVENTIONAL

A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

This clinical trial is investigating a new medication called ALN-APP for individuals diagnosed with early-onset Alzheimer's disease. The main goal is to understand if the medicine is safe and well-tolerated by patients, and how it affects their body. ALN-APP will be given directly into the fluid around the brain and spinal cord, either as a single dose or repeated doses over approximately 12 months. This is a very early stage (Phase 1) study, meaning it's one of the first times this medicine is being tested in humans. The findings will help researchers decide if it's promising enough to continue studying in more detail.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Alnylam Pharmaceuticals

Enrolment target

Start

04 Feb 2022

Estimated completion

20 Apr 2029

Early-Onset Alzheimer Disease

What is this study about?

This study is exploring a new potential treatment called ALN-APP for people who have been diagnosed with early-onset Alzheimer's disease. Early-onset Alzheimer's disease is a form of the condition that typically affects people younger than 65. The medication, ALN-APP, is designed to be given directly into the fluid that surrounds your brain and spinal cord, which is called an intrathecal injection. This method helps the medicine get where it needs to go more effectively.

The main purpose of this study, called a Phase 1 trial, is to find out if ALN-APP is safe for people to take and how well their bodies tolerate it. Researchers will also look at how the medicine moves through the body and what effects it has. They are trying to learn as much as possible about ALN-APP in these first human studies to see if it could be a helpful treatment in the future.

Participants in the study will receive either a single dose of ALN-APP or multiple doses over a period of up to 12 months. By carefully monitoring everyone involved, the research team hopes to gather important information about ALN-APP’s safety profile and its early effects on the body. This information is crucial for deciding if this new medicine should move forward into larger studies.

Key takeaways

This study is testing a new medicine, ALN-APP, for early-onset Alzheimer's disease.
It's a 'Phase 1' study, focusing on safety and how the body handles the medicine.
The medicine is given by an injection into the fluid around the brain and spinal cord.
Participation could last from a single dose up to 12 months.
You might be eligible if you have early-onset Alzheimer's with mild memory problems.
You can stop participating at any time.

Who may be eligible?

This study is looking for adults aged 18 and over who have been diagnosed with early-onset Alzheimer's disease that causes mild memory and thinking problems. You would be considered if your doctor has assessed your thinking abilities and found them to be mildly affected, specifically with a score on a common memory test called the Mini Mental State Examination (MMSE) above 20. Your overall ability to live independently would also need to be mildly affected.

There are certain reasons why you might not be able to join. For example, if your memory problems are believed to be caused by a type of dementia other than Alzheimer's, or if you have specific issues with your liver or kidney function that are outside healthy limits. Also, if you’ve recently taken part in another research study involving an experimental drug, or if you've recently had treatments specifically targeting amyloid proteins for Alzheimer's, you might not be eligible.

The research team will have a full list of things they need to check to make sure the study is safe and appropriate for everyone involved. Your doctor will be able to go through all of these details with you.

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?
Have you been diagnosed with early-onset Alzheimer's disease?
Do you have mild memory or thinking problems due to early-onset Alzheimer's?
Are your liver and kidney test results within healthy limits?
Have you recently participated in another research study with an experimental drug?
Have you recently had other treatments for Alzheimer's that target amyloid protein?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the study medication, ALN-APP, as an injection directly into the fluid around your spinal cord. This is performed by a trained medical professional. Some participants will receive a single dose, while others will receive multiple doses over a period that could last up to 12 months.

Throughout your time in the study, you would have regular visits to the clinic. These visits will involve various assessments to monitor your health and how the medication is affecting you. This includes blood tests, physical examinations, and tests to check your memory and thinking. The first part of the study involves a single dose, and if you are in the longer part, you could be involved for up to one year.

The research team will explain all the procedures, appointments, and the exact timetable for your participation in detail. You will always have the opportunity to ask questions.

Potential risks and benefits

Participating in a clinical study means you could be among the first to receive a new medication, which might help advance our understanding of early-onset Alzheimer's disease. However, as this is a very early stage study, there's no guarantee that ALN-APP will improve your condition, and there's a possibility it may not have any benefit for you. All medications have potential side effects, and since this is a new treatment, not all risks are fully known. The research team will closely monitor your health to identify any potential side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (8)

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

Clinical Trial Site
Verified postcode
La Jolla, United States· Completed
Clinical Trial Site
Verified postcode
Indianapolis, United States· Recruiting
Clinical Trial Site
Verified postcode
Montreal, Canada· Recruiting
Clinical Trial Site
Verified postcode
Toronto, Canada· Recruiting
Clinical Trial Site
Verified postcode
Amsterdam, Netherlands· Recruiting
Clinical Trial Site
Verified postcode
Groningen, Netherlands· Completed
Clinical Trial Site
Unverified
Huntley Street, United Kingdom· Recruiting
Clinical Trial Site
Verified postcode
Sheffield, United Kingdom· Recruiting

Common questions

What is early-onset Alzheimer's disease?

It's a form of Alzheimer's disease that typically affects people younger than 65.

What is ALN-APP?

ALN-APP is a new experimental medicine being tested for early-onset Alzheimer's disease.

How is ALN-APP given?

It is given as an injection into the fluid surrounding your brain and spinal cord.

What does 'Phase 1' mean for this study?

Phase 1 means it's an early study to check if the medicine is safe and well-tolerated in humans.

How long would I be in the study?

It could be a single visit or up to 12 months, depending on which part of the study you participate in.

How to find out more

Alnylam Clinical Trial Information Line

clinicaltrials@alnylam.com 1-877-ALNYLAM Official trial record

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