PI: Peter Pressman, MD, COMIRB# 19-2727
Frequently Asked Questions about the SESAD study:
Am I/is my loved one guaranteed to get the drug/treatment while in the research study?
There is no guarantee to get the drug/treatment while in the research study. However, two-thirds, or 66%, of the participants will receive the study drug, sargramostim, daily via injection. This means that one-third, or 33% of participants will receive a placebo. A placebo is a pill or a liquid given as an injection that looks like medicine but is not a drug. It will have no medical effect on the person that receives it.
How long does the research study last?
This research study is approximately nine and a half months long from start to finish for each participant with a rolling open enrollment of participants over the next several years. This timeline includes around two months to complete the screening process to make sure you are eligible, six months in the treatment phase, and one and a half months for follow-up. It is important that you be able and willing to participate in the entire 9.5-month trial timeframe to measure the impact of your participation.
Can I/we go on vacation while participating in the research study?
You and your study partner need to be available for the appointments that occur during the study period, whether the weekly at-home visits or the occasional visit that takes place at the CU Anschutz Medical Campus. Brief vacations around that schedule can be accommodated if the participant can continue to receive the daily injections and regular safety blood tests.
If the drug is FDA approved, why do you need to do another research study?
We care about your health and safety. Sargramostim is safe and has been approved for over 30 years as a treatment for people who have finished chemotherapy and need to boost their innate immune system. It has not been approved to treat Alzheimer’s disease. We need to conduct this research study to see if the study drug is safe and effective for people with Alzheimer’s disease following an earlier trial that suggested some possible benefit.
Is there an opportunity to continue to receive the drug after my participation or at the end of this research study?
At this time, there is not an option to continue taking the drug after the research study is completed since it is a Phase II trial. The drug would only become available if, and when, the drug is approved by the FDA for use in individuals with Alzheimer’s disease. Learn more about clinical trials here.
If I participate, do I have to come to the CU Anschutz Medical campus every week?
Participants have the option of coming to Anschutz each week to receive study injections, additional supplies for home injections and blood draws, or having a weekly home nurse visit for the weekly procedures and only coming to Anschutz for the screenings and at several intervals during the study as well as the post study 45 day follow up. We do twice weekly blood draw and safety evaluation to make sure you are having no major side effects and that it is safe for you to continue in the research study. Occasionally, there will be other testing that has to be done in person as well, such as cognitive testing and a physical/neurological exam with the study doctor. This is also a chance for our staff to ensure that the medication is being administered correctly at home and review the process with the participant and study partner if necessary. As well, you will have to have the 2nd weekly blood draw either on campus, or at a conveniently located central laboratory facility near your home.
How does the study drug work and what are the potential side effects?
We think the study drug works by activating the body’s innate immune system, allowing the body to work against the Alzheimer’s disease process. Participants in the research study will receive the study drug or a placebo via an injection under the skin five times a week for six months. The trained study partner will administer the injection each day at home, except for the day that they come in to the Anschutz Medical Campus for the in-person visit. Possible side effects from the injection include a reaction and possible infection at the injection site. No serious side effects of the drug were found in the initial three-week trial.
I’m hesitant about the lumbar puncture. Why do you need me/my loved one to do that?
Lumbar punctures provide valuable information for research about what is happening in the brain and are also one of the ways that we confirm a diagnosis of Alzheimer’s disease. Many research studies for Alzheimer’s disease have failed because the people in the study did not actually have Alzheimer’s disease, but another form of dementia. This procedure allows us to be sure that the people included in the study have the disease that we are studying. Possible side effects are discussed in greater detail with the study doctor at the first visit. To learn more about lumbar punctures, visit https://www.coloradoagingbrain.org/importance-of-cerebrospinal-fluid-csf-in-our-research/.
Why are there so many blood draws?
One of the potential effects of the study drug is changes in blood work. We do blood draws twice a week to monitor the bloodwork for participant safety. If the bloodwork showed certain potentially unsafe laboratory values, the study may pause or stop treatment.
What are the roles and responsibilities of the study partner?
Each participant needs to have a study partner who is willing to attend weekly visits, provide daily injections once trained, and to serve as a direct source of feedback as to any changes in the participant’s health and cognitive functioning during the study period. Other family members may participate in visits but a steady study partner is needed to participate in the trial and also needs to provide a written informed consent, in addition to the participant providing written informed consent. The study team will support both the study partner and the participant with trainings, ongoing communications and connections to social supports and community resources that will facilitate study participation.
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