Clinical Trials 101 – Guest Post by Carmen Gonzalez

The world of clinical research can present a hornet’s nest of almost incomprehensible clinical jargon to the average Jane or Joe. Added to that reading burden is finding what study is right for you.  Assuming you have waded through all the public information, there remains the task of asking the right questions so that your experience is productive, worthwhile, and fits your life. In this blog, I will cover “just the basics,” arming you with the essentials to find what you need.

How to Find Them

If you are starting at square one, you probably don’t know how to find clinical studies. First, start by inquiring from trusted sources, including your doctor and other medical professionals with whom you’ve established trust.  If searching for breast cancer-related studies, visit disease-specific sites where patient-centered resources are available which feature clinical studies, e.g. The Susan Love Research Foundation’s Army of Women. Being the empowered patient that you are, expand your knowledge of available studies further by seeking out online clearinghouses, such as BreastCancerTrials, a non-profit dedicated to helping breast cancer patients connect with researchers. BreastCancerTrials.org compiles the trials from ClinicalTrials.gov. and Cancer.gov—comprehensive libraries of ongoing studies— into one hub, so it saves you two less searching chores. There’s an added benefit with BreastCancerTrials.org: they use your health history to guide you to the right trial, and they eliminate a lot of clinical jargon that can trip up novices to clinical research. While you can choose to see all the trials available, their screening tools make it easier to find what you need (see Figure 1).

If you do decide to explore ClinicalTrials.gov directly to explore clinical studies, be forewarned: technical clinical language lies ahead. Search  trials by inserting general terms, such as “breast cancer,” “stage 2,” and “HER-positive.” To tame what could be a fire hose of responses, insert search terms that narrow your focus, such as the disease topic with your location and other distinctive traits, e.g. “Chicago” AND “breast cancer” AND “metastatic cancer.”

Here are a few rules of the road when considering different categories of studies.  Research trials are either observational or investigational, meaning only as to the latter are patients given a treatment. There are four basic types of investigational studies : phase I, II, III, and IV studies. Phase I refers to studies which are being conducted for the first time in humans, having been successfully tested in animal models. Phase I studies are exploring the safety of the drug or device, and are typically involved with  small number of participants. Phase II studies involve a larger group of study volunteers to test if the drug or device is effective, commonly against a placebo. If a drug or device is successful in its phase I and II stages, then a significantly larger study group is involved in phase III to determine if the drug or device is as good or better than the standard of care. During this phase, issues of safety are also closely watched. While success at the phase III stage poises the drug or device for likely market approval by the FDA,  phase IV is imposed to see if late-breaking safety issues arise in the wider marketplace. What does this boil down to for you? The more phases the drug or device has passed successfully, the safer it  has proven to be. Foolproof? No. Remember Vioxx? That’s why Phase IV is so important.

What to Ask About

Whether you are healthy or sick, an appropriate clinical study has to fit into your life for it to be meaningful and worthwhile. That means that you need to have a full understanding of the study’s implications to your health and full knowledge of the obligations before you. Let’s consider these topics separately. First, dig into whether this study offers you significant personal benefit, and if it advances the science in the field. Start by asking how this study advances scientific knowledge and how it improves your prospects of recovering, improving, living longer, or in better managing your disease.
Ask the study team if the research offers a novel treatment, device, or delivery method.  Once you get answers to these questions, mull them over with your doctor if you’re unsure of its potential benefit to you.

Once the overarching considerations meet with your satisfaction, use the following checklist when asking the clinical study team about the study’s logistics:

• Are there any anticipated side effects? What are they and their severity?
• How long is my study obligation? (e.g. number of days, weeks, months, years)
• How many visits in total will require my in-person presence at research clinic?
  • How many visits per week or month does this mean for me?
• Are there additional visits to other specialists beside the research clinic? (e.g. imaging tests at a radiology lab)
  • How far away are these office from the research clinic?
• What other study duties are required of me?
  • Is there a diary I have to keep?
  • Am I required to take study medications at home?
  • Is there a medical device I have to use?
  • Are there dietary or activity restrictions?
• Are there any post-study activities required of me? (e.g. phone check-ins)
• What is the stipend offered for my participation? Is my travel compensated?
• When will the study officially end? When and where can I obtain the study’s results?

Once you inquire about what is involved, now ask about the likely impacts on your family. This is often overlooked and leads to unanticipated conflicts, so tackle them beforehand. While you can walk away from any study at any time, it will save you considerable time and frustration to find the right fit upfront. Consider asking the following:

• Does the schedule conflict with family vacations or other commitments? Is there a way to accommodate these priorities? For example, if study visits are required at a time of day that is inconvenient, will the team consider other time slots or weekends?
• Does the study impose obligations that conflict with your religion? Take the fasting periods of Ramadan for example, and some studies conducted during the summer might not work with your requirements.

Be realistic about where you are in your emotional life as well. If you are undergoing grieving for a marriage or loss of a loved one, you might not be ready to be a study volunteer. Acknowledge your own expectations and biases regarding the study and discuss those reservations and hopes with the study team. They can provide able counsel to help you determine if participation makes sense at this moment.

What To Expect

If you are placed in a control group—study volunteers who are not receiving the active study medication or device—then any positive response you experience will be attributed to what is called the “placebo effect.” You won’t know whether you were assigned to a placebo or “active” group, as that would defeat the scientific process. However, sustained dramatic improvement tends to suggest that patients were in the “active” group. For patients to gain answers to which groups they were in and the overall impact of the study, they must wait for the trial’s conclusion. In the U.S., study teams are required to post their results on ClinicalTrials.gov upon completion, but faithful adherence is spotty at best. To ensure you get the answers you need, aside from your personal outcome, request the results from the study team and create a tickler on your calendar to remind those in charge to keep you posted.

If during the course of the study, you experience unwanted side effects, immediately report your symptoms to the study team right away for intervention. Depending on the study, there are some minor  side effects that are anticipated, but in all cases, report your experience no matter what. For patients with multiple pre-existing medical conditions, there may be fewer studies for which they are eligible, but that rarely rules out everything.

In studies that span several months or years, the mere slogging pace of the study can impose a weariness commonly called “study fatigue”—not an ailment, but an acknowledgement of growing impatience. Be honest with yourself if you can endure a long-term study and the duties that come with it. While you can’t predict all the events that might upset your study schedule commitments, understanding and knowing how you manage everyday routines will give you a candid idea of your likely study visit consistency. Armed with these tips, you can make better decisions concerning study involvement.