Frequently
Asked
Questions

Yes, you can change your mind at any time before the actual investigation has begun. During the intake interview, we inform you about what you can expect and help you make up your mind.

Yes, for TRACK DCAA, the minimum age is 25 and the maximum age is 60. However, it is very important that as many people as possible register to participate in research, even if there is no ongoing research you are eligible for right now. You can register from 18 years and upwards.

The RNA therapy Mivelsiran that is developed by Alnylam is currently being investigated in a phase II trial in sporadic CAA patients and will soon be open to DCAA patients and genecarriers in Perth as well. We expect that enrollment will start early 2025. 

If you have any doubts about something or you are worried, you can either talk to Sanne van Rijn, who is a Psychologist and patient advocate who comes from a DCAA family herself, or talk to Samantha Gardener, a scientist who has been part of the DCAA research team for many years.

Yes! You can participate in TRACK DCAA, even if you do not know whether you carry the gene. We make sure there is no way you can find out through the study whether you are a gene carrier. If you don’t want to know, you will never find out through participating in TRACK DCAA. If you do want to know however, we can help you with the process. For the trial cAPPricorn-1, only DCAA family members who have been tested and know they carry the gene, or who are symptomatic, may enroll.

Currently, we are talking to Alnylam about expending TRACK DCAA and continue enrolling family members. Right now, there are no new participants enrolled in the study. When you want to participate in the trial, there will be a screening assessment, to find out whether you fit the criteria of the study. If you want more information about what the screening assessment entails, please contact the research team.

You can participate if you are a DCAA gene carrier, have a 50% chance of carrying the gene because one of your parents does, or a 25% risk of carrying the gene as one of your grandparents does and the genetic status of your parent is unknown. Anyone over the age of 25 and under the age of 60 is eligible for TRACK DCAA, who hasn’t had more than one symptomatic stroke. If you are not eligible now, but you want to participate in the future, please still contact us. We would love to be able to get in contact with you when the opportunity for future research emerges.

The research team can either book your accommodation for you or refund accommodation costs to a reasonable nightly rate. Petrol and parking costs are also refunded by the team.

No, absolutely not. It remains your choice whether to make that information public, or not. When you decide to participate, all information about you is stored completely securely. This is required by law. Only members of the research team have access to your information and they work according to ethical and legal regulations.

If you are still eligible for TRACK DCAA and/or cAPPricorn-1, depends on your disease progression. We are happy to have a conversation and together find out what we can mean to each other. Do not hesitate to contact us, even if you are not eligible for TRACK DCAA, we would love to know that you are up for participation if and when the opportunity arises.

Dutch and Australian family members have been participating in natural history studies for many years. The studies have shown that especially in the cerebrospinal fluid taken by a lumbar puncture, we can already see a change in amyloid clearance from the brain from late 20s / early 30s in gene carriers. Obviously this is something that might scare us as family members, but at the same time this knowledge helps us to intervene as early as possible. This is the reason why we ask young people, who are not personally affected by DCAA (yet), to participate. You can join in if you do not want to know your genetic status, so you can safely participate without finding out if you are a gene carrier.

No, you cannot, they have to sign up themselves. We encourage you to have a conversation about research and DCAA, but they must make a choice themselves.

We cannot give an absolute number. The more people participate, the greater the chance of success. The widest possible group in terms of age and gender helps enormously to learn about disease development in the brain. As of July 2023, 24 people from different DCAA families in Australia participate in TRACK DCAA. We are very thankful to them and hope that more of you choose to do so.

We are hopeful, but we know that drug trials can fail. However, being part of a drug trial does mean that you will have access to a potential drug. If it works, this means that we slow down the accumulation of amyloid in the brain and therefore slow down the disease trajectory in gene carriers.

We obviously hope so! But the honest answer is: we don't know. Drug development takes many years. Yes, that hurts, we know. So why should you participate in research? You help anyway! If it's not for our generation, then for our children, nieces and nephews.

We do everything we can together to make that happen. But drug development is a complicated process that takes time. We are positive about a number of potential drugs, but we have to be realistic. It may take a number of years before there is a treatment for Dutchtype CAA. Even if it may be too late for you, your participation means helping all of us towards the development of a drug.

It depends on the drug. When you are thinking about participation, you will have a lengthy conversation with somebody from the research team. They will inform you about pros and cons and help you decide on participation. Moreover, a drug company will NOT be granted permission for a drug trial if the risks outweighs the rewards. This is assessed in advance by national regulators and the Medical Ethical Review Committee.

The study helps us understand more about how the disease develops in the brain of gene carriers from a young age. We hope that the study helps us get into drug trials. When you do a drug trial, you want to know two things:  1) when is the best time to start the intervention? and 2) how can we measure if the drug actually slows down the disease? Basically, you need to know what is the best way to see how Dutchtype CAA behaves in the brain and if that changes when you use a potential drug. TRACK DCAA helps us understand if the methods we think do that, actually do. That is necessary if we want to be part of drug trials.

The research team hires specialists to take you through all parts of the study safely and professionally. Because not all of them are available on the same day, unfortunately we have to schedule your appointments over a period of time.

Your first contact is with Samantha Gardener, PhD, who is part of the research team at the research centre in Perth. She talks you through what to expect and answers all of your questions. You then get a consent form containing all the information you need to know before participating and when you agree, you sign. This is followed by a clinical assessment with Doctor Ana Gnjec, and fasting blood samples.  This is followed by memory testing and the It’s the Clinical Dementia Rating either with Clinical Neuropsychologist Registrar India or Vandhana. PET-scans are done on Thursday at Oceanic Medical Imaging at Hollywood Private Hospital. The MRI-scan is on Saturday at SKG Radiology at Hollywood Private Hospital. Your visit ends with undergoing a lumbar puncture in the research centre on the Tuesday morning.

During a PET-CT scan, you will receive a ‘tracer’, a slightly radioactive glucose (sugar) through an IV. This tracer moves to your brain, where it sticks to the toxic amyloid deposits that cause DCAA. You will then undergo a brain scan, a PET-CT scan. This method helps make amyloid deposits in the brain of gene carriers visible. The method has been used in Alzheimer’s research by the Perth research team for a long time and is safe. It is a relatively new method for Dutchtype CAA. At the moment, the PET-CT scan is therefore less informative than, for example, the lumbar puncture. Part of the goal of TRACK DCAA is to examine whether the PET-CT scan is an effective way to visualize the course of the disease.

A small amount of  a tracer that sticks to amyloid in the brain is administered through an IV. The substance is not dangerous and disappears from the body through urine within a few hours. The tracer has no side effects. Once the tracer has been administered, you should wait a while so that your body has time to absorb it. During this waiting period you have to rest, but you can, for example, read a book or magazine or use other media, such as watching a movie on your phone.

If the substance is sufficiently absorbed, both a PET scan and a CT scan are made. It is important that you lie still during the PET-CT scan, which is why there will be a band around your head to support you. The computer then combines the results of the two scans. This makes it easier to see where the tracer is absorbed in the brain and helps to locate amyloid deposits. The scan takes up to a total 60 minutes. The entire examination, from administration of the substance with the waiting period to the end of the scan, takes about 2 hours.

In the PET-CT scan we use radiation and a radioactive substance. During TRACK DAA you receive 20.1 mSv. In comparison: the background radiation (the radiation that one incurs from natural radiation sources in the environment) in Perth is about 2 mSv each year, in the Netherlands it is about 2.5 mSv per year. People in Switzerland even receive about 5.8 mSv of natural background radiation per year due to the altitude. We recommend telling us if you are undergoing any other therapy that involves radiation at the same time. It is highly unlikely that the radiation from the scan in this study harms you, however, we advise you not to participate in any other scientific studies involving radiation at the same time. You will still be able to undergo an examination or treatment with radiation for medical reasons.

Participants who take part in TRACK DCAA undergo an MRI, a PET-CT scan, a lumbar puncture and cognitive tests. We do not take asking these things of you lightly. Together we decided that these parts of the study are necessary, because we want to get a clear understanding of what happens in the brain. An MRI helps us picture (micro)bleeds, a PET-CT scan makes amyloid deposits in the brain visible, a lumbar puncture shows us how much amyloid is ‘cleared’ (or not cleared) from the brain through the cerebrospinal fluid (CSF) and the cognitive tests show us the effect of the disease on attention and memory. You will also undergo a clinical assessment and will be asked to give blood. Both blood and CSF are checked for biomarkers – indicators of disease.

With a lumbar puncture (also called a spinal tap) a small amount of cerebrospinal fluid is taken from your spine. This fluid normally sits around the brain and around the spinal cord. It is there for protection, but is also a clearance system for the brain. The lumbar puncture is performed in a safe manner by an experienced anesthetist. In total, the lumbar puncture procedure takes about 10 minutes, then you remain lying down for a number of hours to reduce the chances of post procedure headaches.

Logically, the lumbar puncture is not the part that people most look forward to. We would not ask you to take part in it, if it wasn’t safe and very important. By means of the lumbar puncture, cerebrospinal fluid is taken from your spine with a narrow needle at the bottom of the spine. Cerebrospinal fluid surrounds the brain and serves as protection, but also as a "waste system". In the collected cerebrospinal fluid, the researchers can see how much of the toxic amyloid protein is or is not removed from the brain in gene carriers. This method is currently the only way to visualize the protein directly and therefore very important. It will most likely also be the most reliable way in future drug development to be able to measure whether a drug does what we hope it does: ensure that less amyloid clutters the brain. During cAPPricorn-1, the drug Mivelsiran is administered through a LP as well.

The examination with an MRI scan takes up to 60 minutes. In the MRI scan, which looks like a deep tunnel, you are lying on a bed. You won't feel anything during the scan. There are no risks involved. When sliding in and out of the MRI scanner, about 1 in 3 people experience dizziness, which usually always disappears within a minute. The scanner makes quite a lot of noise, so to protect your hearing we provide you with earplugs. During the scan you can listen to music, possibly a CD you brought yourself. In addition to the regular MRI scans, a functional MRI (fMRI) is also being done. This means that while you are in the scanner you will look at a moving picture for about 10 minutes, which helps us study blood flow in your brain.

An MRI scanner is a kind of tunnel with a bed in it. It makes use of a magnetic field and radio waves to take pictures of your brain. This method is completely safe. The photos give us a picture of bleeds and microbleeds in the brain of gene carriers. This gives us more information about from what age approximately, and how, the disease develops in the brain over time.

TRACK DCAA is a natural history study. This means that we are studying the course of the Dutchtype CAA in gene carriers. TRACK DCAA is sponsored by two pharmaceutical companies: Alnylam and Regeneron. The aim of the study is twofold: 1) learning more about from what age the amyloid starts to clutter in the brain and 2) learning more about how to measure the progression of the disease in gene carriers. Studying these things is necessary for a potential drug trial in the future. TRACK DCAA helps gain insight as to what age to start an intervention that hopefully slows down the disease, and also helps us understand during a drug trial how to measure if a drug is actually slowing down the disease.

Yes, please! We want to know more about how the disease develops way before there are symptoms. If you are over the age of 25, we would love for you to be part of TRACK DCAA. When you are a known genecarrier and over 30 years old, you are also eligible to participate in the trial cAPPricorn-1.

Because it is so important that people who have indicated that they do not want to know whether they are gene carriers cannot accidentally find out, it is not possible to find out through the study if you are a gene carrier. However, if you want to, your brain scans are available to our neurologist Doctor Daniel Clarke. He is available for appointments every Monday afternoon and specialized in genetic and sporadic CAA. The team can also help you undergo genetic testing to find out your genetic status, if you wish to. You can find out more about genetic testing here (link to www.hchwa-d.nl/en/).

We understand this question very well, and yes, you could. But that would be a real shame. Because the number of people participating in TRACK DCAA, help us design the drug trial we are desperate for. Because DCAA is so rare, as family members we really need to work together.

We are able to enrol participants in TRACK DCAA who may be planning on getting pregnant during the two years of the study. You will need to complete the whole assessment at baseline so cannot be pregnant at this assessment point, but if you are pregnant at 12- or 24-month assessments we will only complete the procedures which are safe to do so. For trials it is usual to exclude female participants who are planning to get pregnant or male participants when their partner is.