Frequently Asked Questions about Phase I Clinical Trial: an investigational vaccine for treatment of Alzheimer's disease in people with Down syndrome.
If we want to sign up, what do we need to do next?
The next step will be to complete this online eligibility survey.
Why is this research being done?
In this study we will be looking at an experimental vaccine (sometimes called study drug or investigational drug) that we call ACI-24. A vaccine is an injection that is designed to help your body prevent different health and medical conditions that might make you sick. The experimental vaccine that we are studying has not yet been approved by the Food and Drug Administration. The purpose of this study is to see if it is safe and to see if it works.
What age must the person with Down syndrome be to participate in this study?
The person with Down syndrome must be between the ages of 25 and 45 years to be eligible to participate in this study.
How many people will be in the study?
There will be about 24 participants in the study from around the world, and we can enroll up to 5 here at Massachusetts General Hospital. The study will be conducted at several different locations in the USA.
Will I be paid? Will my expenses be covered?
Parking will be validated. The person with Down syndrome and their accompanying caregiver will each be compensated $75 for their time for the completion of each study visit. The full study has 22 visits. A gift card of $30 for lunch will be provided for each study visit. Additionally, the caregiver will receive $25 per visit to help offset travel costs.
Will being in this study help my family member with Down syndrome?
There is no guarantee that your family member will benefit from this study. Information from the study might help researchers to come up with new tests or medications to help people with Down syndrome in the future.
How do I know if my family member with Down syndrome is eligible to participate?
Please review this online eligibility survey to help determine who is eligible—and who is not—to participate in this clinical trial. These criteria were established by the study Sponsor. The person with Down syndrome does NOT need to be an established patient in the MassGeneral Hospital Down Syndrome Program in order to participate in this research opportunity.
Please remember, people will be considered on a first-come/ first-serve basis. At MassGeneral Hospital, we will have up to 5 spots for those who are interested and qualify. MassGeneral Hospital may be granted additional subjects to enroll based on the enrollment numbers at other centers.
How long does the study take?
The study involves 22 visits over 2 years. A consistent caregiver will need to accompany the person with Down syndrome to each appointment.
What does this clinical trial involve?
Each participant in the study will be given either the study vaccine or a compound that contains no medicine (placebo). A placebo looks the same as the study vaccine but has no medicine in it. Neither you nor the study doctor will know which drug is given. If you agree to take part in this study, you will be asked to make 22 clinic visits over a period of 2 years. These visits will last between 1-8 hours.
How do you get the experimental vaccine?
The experimental vaccine comes in little syringes (shots) that have small short needles and will be given in 1 or 2 doses each time you come to the clinic. The nurse practitioner will help you chose 2 places on your leg for the shots and will give them when you are ready. You will only get the vaccine injections on 7 of the 22 study visits at the clinic.
Will any part of the study hurt?
When the nurse practitioner takes blood, the person with Down syndrome may feel a little pain from the needle prick, but the pain will go away. We might have to try again to make sure it is done right. They may feel dizzy or have a bruise, but this won’t last long.
When the recording of your heart (ECG) is done, it is possible that the pads may cause some itching and redness where they were stuck to the body.
It is possible that the person with Down syndrome will feel tired during the study visits because there will be several different tests in one appointment. Participants will be able to take breaks if needed.
Some people get anxious when they lie in the MRI machine. This is because it is a small and noisy space. Some people do not like the loud sound that the machine makes when it is turned on and working.
The investigational vaccine that is being tested is new. It may or may not make the person with Down syndrome feel sick. The investigational drug may or may not make them feel better.
What are the good things that can happen from this research?
The investigational vaccine being tested is new, so we don’t know yet if it will help people with Down syndrome. When we finish the research we hope that we will know more about whether it may help people who have Down syndrome.
Who will know that you are in the study?
Participation in the study is kept confidential. When the study is finished, we will write about what we found out for all participants in this study. We won’t use your name. In case of a medical emergency, a doctor or nurse practitioner and some people from the clinic will be allowed to see your name and information from your tests. But nobody else is allowed to see your personal information.
What are your rights as a research participant?
Being in this study is voluntary. You don’t have to be in this study if you don’t want to or you can stop being in the study at any time. Your decision will not result in any penalty or loss of benefits that you have now. If you have questions about your rights you may call:
Partners Human Research Committee office at 857-282-1900.
You will be told about any new information that may affect your health, welfare, or willingness to stay in this study.