Clinical trials are research experiments meant to answer specific questions about new treatments including medications. They are tests of potential treatments, usually drugs but also medical devices and therapies, on human volunteers.
These 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 to get that approval is by proving 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, while not common, do exist.
The reasons a loved one with Alzheimer’s or other dementia might participate in clinical trials include:
– Access to the newest drugs that might slow or hopefully even 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 study doctors and nurses may be higher than from personal doctors.
– Trials are often paid (see below).
– Help in the fight against devastating illnesses including Alzheimer’s and dementias including vascular, Lewy body, frontotemporal and even mild cognitive impairment (MCI).
There are risks as well, which include:
– Existing treatments may be better than the experimental treatments given to trial participants.
– Experimental treatments might work for some people but not others.
– Though agencies are monitoring clinical trials to ensure safety (see below), unexpected side effects may occur, particularly during Phase 1.
– Though participants are usually compensated, the full costs of joining a trial (like transportation) may not be covered.
A long and layered process is meant to ensure that clinical trials are safe for every participant. It starts with a goal: Create a compound that will change the body in a way that helps fight or eradicate illness, without causing harm. Hundreds or even thousands of compounds with slight variations are created and tested on cells kept or grown in a laboratory. (These are test-tube experiments called “assays.”) Computer simulations are often also part of these early steps.
If a compound shows potential, it’s on to the next phase, which is usually animal testing. Scientists try to use as few animals as possible, but at least two species are experimented on because drugs may interact differently with different animals. Measurements are taken, such as absorption into the bloodstream and how the compounds are broken down. For medical devices, they need to determine whether the device works when interacting with living tissue.
Only about one in 1,000 compounds make it through animal testing to human trials.
All this to say: Trials are as safe as they can possibly be before they get to the human testing phase, when your loved one might take an experimental medicine. The FDA says this is the reason for animal trials: A treatment must be proven safe before it can be put in or on a human being.
Further, an FDA Institutional Review Board (IRB) is tasked with monitoring clinical trials. IRBs are composed of qualified doctors and scientists whose job is to assure in advance, and then via review during the study, that participants are safe and treated respectfully. The IRB also inspects study sites, where the trials are held, and interviews researchers conducting the review.
Here’s what family members need to know when a loved one signs up to join a clinical trial:
– Participants have the right to leave any time.
– Absolutely all 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 should have been reviewed by the FDA to make sure they are completely lawful. FDA IRBs also check for readability, so an informed consent form isn’t written in such complicated terms that it’s hard to understand.
Companies conducting clinical trials must follow a written plan called a “protocol.” The protocol usually includes the following:
– The trial’s objective, or what researchers hope to prove. For readers of Dementia Care Central, that probably means improving symptoms from dementia.
– Design and methods, or what the process will be for participants to try the treatment and be tested for its effects.
– The length of the study.
There’s no standard length for clinical trials. They could be months or years. Typically, if participants are testing a single-dose medicine to relieve symptoms, or some short course of treatment, then it may not be very long. But for preventative treatments or to study a drug’s effect over multiple stages of dementia, the clinical trial could take years. That said, regulations are so strict that by the time an agency is seeking participants, anyone who agrees to sign informed consent documents should have an idea of 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 (could be a single dose or several doses over a very long time), while 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’s a difference. Participants don’t know whether they’ve been given the new treatment or the existing / placebo treatment, because “blinding” (as this is called) ensures there’s 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 often broken into phases, defined by the FDA:
– Phase 1: Testing for safety, the Phase 1 trials often administer very low doses to monitor reactions including side effects and how a drug is absorbed by the body. Typically, less than 100 people participate in Phase 1.
– Phase 2: Many more people (hundreds) will participate in Phase 2, if no harmful effects were found in Phase 1. In Phase 2, safety and side effects continue to be monitored, but researchers will use 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 might receive the drug or treatment. Whole populations can be studied, for example to see whether people in different parts of the world react differently. Larger doses may 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 for the FDA for approval, and then marketing might begin.
– Phase 4: Studies the new drug or treatment after it’s gone out into the 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 further comparison with other drugs or treatments and determining whether there are longer-term side effects.
Not all clinical trials pay money to people who sign up, but there is often financial compensation for participants. Additionally, money for travel, expenses, and lost wages may be available. What to know about getting paid to participate:
– Phase 1, with fewer participants and a higher risk of unexpected side effects, usually pays the most. The amounts vary hugely, but compensation can be in the thousands.
– Later phases may not pay at all – the potential health benefits are the incentives. If they do, it’s likely a few hundred dollars (though this, again, varies hugely).
– Participation in clinical trials should not be considered a paying job, despite the compensation, but there may be more work than just taking a pill and being monitored. 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 me” is the National Institute on Aging’s search tool for dementia-related criteria here. Considerations you’ll want to weigh include location and timing. Participants must be diagnosed for a specific type of dementia such as vascular, frontotemporal or Lewy Body dementia and 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 some number of weeks or months before the trial.
Be prepared for trials to be very time-consuming, often requiring detailed assessments of your loved one over many weeks. Researchers may, for instance, need to make frequent blood draws to test for concentrations in the blood. If they’re trying to determine whether a compound is improving symptoms, then assessments will be necessary. Other tests, like an MRI, are also usually part of the trials process. Caregivers may be responsible for transportation, medication management, and more, so make sure you have a complete understanding of the process before signing up to join a clinical trial.