Clinical trials are research experiments that scientifically prove if a newly developed treatment is effective. These potential treatments are usually in the form of medication but there are also tests for medical devices and therapies.
Clinical trials are necessary for creating medications to treat illnesses. The U.S. Food and Drug Administration must approve a medicine for treatment before it can be made available to the public. The only way for this to happen is for drug makers to prove its effectiveness and safety through clinical trials.
Most participants in clinical trials reside at home. Clinical trials in assisted living, memory care, or long-term care residences such as nursing homes, are not common but do exist.
There are many beneficial reasons your loved one with Alzheimer’s or another form of dementia might participate in clinical trials. They can include:
– Access to cutting-edge drugs that might slow or hopefully reverse the progression of the disease. Participants are among the first to see the benefits of treatments.
– New therapies might improve the participant’s emotional well-being.
– Attention and quality of care from the study’s doctors and nurses might be greater than from one’s personal doctor.
– Trials are often paid (see below).
– Help fight Alzheimer’s and other forms of dementia through one’s participation to create more scientific research.
There are risks which include:
– Existing proven effective treatments can sometimes be safer than experimental treatments given to trial participants.
– It can be frustrating that some experimental treatments work for some people but not for others.
– While monitoring clinical trials to ensure safety (see below), unexpected side effects may occur. This can be especially true during Phase 1.
– Participants are usually compensated, but the extra costs of participating in a trial (like transportation) may not be covered.
Clinical trials have multiple checks and balances meant to ensure the safety of participants. It begins with creating a compound that will change the body to help fight or eradicate an illness, without causing harm. Thousands of compounds with slight variations are created and tested on cells in a laboratory. These test-tube experiments are called “assays.” Computer simulations are often used during these early steps.
After a compound shows potential, it can pass to the next phase, usually animal testing. Scientists try to use as few animals as possible, but at least two species are experimented on. That is because drugs may interact differently with different animals. Additionally, the FDA says that “a treatment must be proven safe before it can be put in or on a human being”. Data is recorded, such as absorption into the bloodstream and how the compounds are broken down. Medical devices need to prove whether the device works when interacting with living tissue. Only about one in 1,000 compounds make it through animal testing to human trials.
The result is that trials are as safe as they can possibly be before they get to the human testing phase. This is the step when your loved one can be a candidate for an experimental medicine.
An FDA Institutional Review Board (IRB) is tasked with monitoring clinical trials. IRBs are made up of doctors and scientists whose job is to make sure ahead of time and during the study that participants are safe and treated respectfully. The IRB also inspects clinical trial sites and interviews researchers who are conducting the experiments.
There are a couple of key pieces of information to keep in mind when a loved one signs up to join a clinical trial:
– Participants can leave the study at any time.
– All potential risks must be explained by researchers to trial participants before the study begins.
Before becoming part of the trial, your loved one will sign informed consent documents. These documents are reviewed by the FDA to make sure they are lawful. FDA IRBs also check for readability, making sure informed consent forms are not written in complicated terms that are hard to understand.
Companies conducting clinical trials must follow a written plan called a protocol. Protocols generally include:
– The trial’s objective, or what researchers hope to prove. In terms of the scope of this article, it means improving your loved one’s symptoms and quality of life because of dementia.
– What the process is for participants when they begin treatment and how its effects will be tested.
– The length of the study.
There’s no standard length for clinical trials. They can run for a couple of months or a couple of years. Typically, if participants are testing a single-dose medicine to relieve symptoms, a study might not be very long. For preventative treatments or those studying a drug’s effect over multiple stages of dementia, a clinical trial could take years. Regulations are strictly set making it so that by the time the study is looking for participants, anyone who agrees to sign informed consent documents will know how long the trial will last.
Often, clinical trials are comparing groups. Participants who all have the same illness will be split up. One group is given the experimental treatment. This could be a single or several doses over a long time. The other group is given an existing treatment or a placebo (like a sugar pill). The effect on each group is then measured to determine whether there is a difference. Participants do not know whether they have been given the new treatment or the existing / placebo treatment. This is because what is referred to as a blind study ensures there is no bias in the results.
There will probably be an amount of time after the trial when researchers are following up with your loved one to determine longer-term effects. This can be weeks, months, years, or even decades.
Clinical trials using human participants are broken into phases. The FDA has defined these as:
– Phase 1: Testing for safety, Phase 1 trials often administer low doses to monitor reactions. This includes side effects and how a drug is absorbed by the body. Typically, less than 100 people participate in Phase 1.
– Phase 2: More people (normally in the hundreds) will participate in Phase 2. If no harmful effects were found previously, in Phase 2, safety and side effects continue to be monitored, but researchers will focus on control groups to determine effectiveness. This usually means that one group of people will be given the experimental drug or treatment, while another group is given an existing drug or a placebo. The reactions are then compared.
– Phase 3: Large-scale assessment, when thousands of people can receive the drug or treatment. Whole populations can be studied to see whether people in different parts of the world react differently. Larger doses can be tested to determine how much should be prescribed. Experimental drugs might also be combined with other existing drugs during Phase 3. After this phase, the drug is fully submitted to the FDA for approval, and then marketing might begin.
– Phase 4: Studies the new drug or treatment after it is being used by the general public, and will assess more than 1,000 people to gauge their reactions. This is when cost-effectiveness for patients can be assessed, as well as comparisons with other drugs or treatments to determine if there are long-term side effects.
Not all clinical trials pay money to people who sign up, but it is normal for there to be financial compensation for participants. Additionally, money for travel, expenses, and lost wages may be available. Before participating, consider:
– Phase 1, with fewer participants and a higher risk of unexpected side effects, this stage normally pays the most. The amounts vary, but compensation can be in the thousands.
– Later phases may not pay at all – the potential health benefits are the incentives. If they do pay, it is likely a few hundred dollars (though this, again, varies hugely).
– Participation in clinical trials should not be considered a paying job, despite the compensation. Clinical trial participants may be asked to keep detailed records of their experience, and tests might be long and strenuous. Given persons with Alzheimer’s or dementia may not be able to keep detailed records, this task may fall on their primary caregivers.
– Travel costs might be covered for participants, but this is not always the case.
– Payments for participation are taxable if they’re larger than $600, though this does not include reimbursements for expenses like travel. If the organization running the trial pays more than $600, your loved one should receive a 1099-MISC document (for miscellaneous income) to report the money to the IRS.
A good way to find clinical trials near you is through the National Institute on Aging’s search tool for dementia-related criteria. Click here for more information. You will want to consider location and timing. Participants must be diagnosed with a specific type of dementia such as vascular, frontotemporal, or Lewy Body dementia. Additionally, it may matter which phase of the disease your loved one is in. There may also be a requirement to get off certain medications for a certain number of weeks or months before a trial begins.
Be prepared for trials to be very time-consuming. They often require detailed assessments of your loved one that are done over a period of many weeks. Researchers may need frequent blood draws to test for a drug’s concentration. If a drug maker is trying to determine whether a compound is improving symptoms, then assessments are necessary. Other tests, like an MRI, are also usually part of the trial process. Caregivers may be responsible for transportation, medication management, or other aspects of the process. Make sure you have a complete understanding of the process before signing up to join a clinical trial.