A phase 1/2a study of ARO-MAPT-SC in people with early Alzheimer’s disease

What is the aim of the study?

This study is done to test an experimental drug for the treatment of Alzheimer’s disease. ARO-MAPT-SC is an experimental drug which means that it has not yet been approved by a drug regulatory authority.

Alzheimer’s disease is the most common type of dementia, a condition that affects how people think, remember, and learn. People with Alzheimer’s disease gradually find it harder to remember things, solve problems, and do everyday activities on their own. Scientists have found that in people with Alzheimer’s disease, certain proteins build up in the brain. One of these proteins is called tau. The tau forms twisted tangles.

These tangles damage the brain’s nerve cells, called neurons, making it harder for the brain to work properly. People who have more tau protein in their brains often have more serious Alzheimer’s disease symptoms.

There is currently no cure for Alzheimer’s disease and no approved treatments that directly target tau, highlighting the need for further research. Your participation could help advance better treatment options for early Alzheimer’s disease.

ARO-MAPT-SC (the study drug) is being developed to try to prevent, or potentially reverse tau protein build up in patients with early Alzheimer’s. This study compares the study drug (ARO-MAPT-SC) to a placebo (dummy drug).

The aim of this study is to test how safe the study drug is, and also to find which dose is most tolerated in people with early  Alzheimer’s disease.
 

What will participants be asked to do during the study?

People in the study will be put into their treatment groups by chance, like flipping a coin. This is a process called randomisation. There are 2 treatment options that a person could be given. These are the study drug (ARO-MAPT-SC) or a placebo (a dummy treatment that has no medicine in it).

In this study you have a 75% chance of getting the study drug and a 25% chance of getting the placebo. In other words, you are 3 times more likely to get the study drug than the placebo.

Find more information about why studies use blinding, randomisation and placebo controls.

The study medication will be given by injection under the skin (subcutaneous) as decided by the Study Doctor, usually the tummy.

During the study you will:

• have some laboratory tests, such as blood, urine or spinal fluid tests. These tests help doctors check for any new side effects, such as symptoms or health changes that might happen after receiving the study medicines or placebo
• have heart scans called ECG traces
• complete tests that look at memory, thinking, and emotional wellbeing.

Your total participation will be approximately 46 weeks and will include 14 clinic visits. One of the visits may include an overnight stay. Visits are broken down into:

• screening period (before treatment): 8 weeks
• treatment period: 38 weeks

If participants would like more details about the tests, procedures, or number of visits, the study team can provide full information.