What Is A Clinical Trial?

Paul Newhouse, M.D. for CareLiving.org

Developing new and better treatments for Alzheimer’s disease and pre-Alzheimer’s conditions is one of the most important priorities of research scientists and physicians. While there are many steps to discovering and developing new medications (which I will cover in subsequent blog posts), the most critical step of the entire process is performing what is known as a “clinical trial”.

Paul Newhouse, M.D. Director, Vanderbilt Center for Cognitive Medicine
Paul Newhouse, M.D.
Director, Vanderbilt Center for Cognitive Medicine

A clinical trial is, at its simplest, a test of a new or novel treatment, medication, or combination of medications to evaluate whether it improves the signs or symptoms of the disease. Large-scale clinical trials (so-called “pivotal trials”) are what the FDA uses to decide whether to approve a medication for sale in the United States for a disorder like Alzheimer’s disease.

When we think of clinical trials in Alzheimer’s disease, we are usually adding on a new treatment to the already approved medicines that the person may be taking, to see if the combination improves the symptoms or course of illness more than the standard treatment alone (so-called “add-on” trials). Many trials also involve some patients taking a placebo medication that is designed to look exactly like the active medication, but is not thought to have the same effect. This is necessary to separate the effects of the new medicine from the effects of simply being in a clinical trial. For pre-Alzheimer’s conditions such as mild cognitive impairment (MCI), there is no FDA approved treatment, so these studies are testing a new medication against placebo.

How long are most clinical trials?

Trials usually last from 24 weeks to 2 years. This process is necessary as treatment effects often take time to emerge, and it may take time for the new medication to show benefits. These days, trials are also checking many biological measures of disease including brain scans, blood tests, and spinal fluid as well as genetic testing. It is our hope that some of these measures may help us look for additional ways that medicines may change the course of Alzheimer’s disease.

Why should I (or my loved one) consider joining a clinical trial?

The advantages of joining a clinical trial for you or your loved one are several. First, doctors will perform a very detailed and careful assessment to help confirm the diagnosis giving new information that may help prognosis and treatment. Secondly, follow-up will be very detailed and you will be offered contact with a supportive and caring team. Third, you will have the chance to receive a state-of-the-art medication that may improve the course of this devastating illness. Finally, the opportunity to help those who come after us by participating in trials is a very important motivation for many patients and families.

We cannot test the clinical benefit of medications in animals, as Alzheimer’s disease is uniquely a human illness. Thus, clinical trials are absolutely critical to advancing progress. Joining a clinical trial could be one of the best investments of time that you or your loved one ever makes. Learn more about clinical trials in general and what trials our center is offering at the Vanderbilt Center for Cognitive Medicine (medschool.vanderbilt.edu/ccm/).

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