Clinical Trials 101 - Your Guide to Lung Cancer & Research

Making decisions can be tough, especially when they relate to your health! One decision you may face is whether or not participating in a clinical trial is right for you. There are several types of clinical trials, and each have different goals and requirements. So, while one clinical trial may not be a good fit for you, there may be others that are. If you’re thinking about exploring clinical trial options but still aren’t sure, ask yourself some questions to determine if a clinical trial might be right for you: Am I willing to work with a new healthcare team during the trial? While you can still see your doctor while enrolled in a clinical trial, you will also be seeing a new group of healthcare professionals who are working on the study. Clinical trials are conducted by a group of researchers. The lead researcher for a trial is called the principal investigator (PI), who is usually a medical doctor or other healthcare professional. The research team may also include other doctors, nurses, and researchers. This research team is usually different from the healthcare team you see for your regular care. It’s important to inform your usual healthcare team of the clinical trial you are thinking of participating in and to discuss how the process will work. Am I willing to follow the trial protocol? The clinical trial protocol is a detailed plan of what the trial will involve. When you agree to participate in a clinical trial you will sign an informed consent document, which outlines the protocol and any tests, procedures, or appointments that are expected of you. These expectations may impact your day-to-day life. When reviewing the trial protocol be sure to note when you will need to travel to appointments and how often you may be required to have tests or procedures, like blood draws or MRI scans. You may also be asked to stop taking medications you are currently on while you are in the clinical trial. Keep an up-to-date list of all medications, including over the counter medications, and show this to the clinical trial team to be sure you can continue taking them during the trial. Am I okay with potential unknowns? Some clinical trials are blinded, or masked, meaning that participants will not know what medication they are receiving. Some studies are double-blind studies, meaning both the participant and the research staff will not know what medications a participant is on. This is done to prevent any bias or influence and to allow the participant and researchers to measure the true effect of a drug. In blinded trials, you will be informed of all possible treatments that will be part of the trial, including no treatment (placebo). Before agreeing to participate, it is important to ask yourself if you would be okay with receiving any of the possible treatments, and if you could handle not knowing which treatment you may be given. Am I okay with the possible risks? The research protocol and informed consent document will outline your rights as a clinical trial participant. This document will note the potential benefits for participating in a clinical trial and will also note potential risks. Clinical research is approved and monitored by an Institutional Review Board (IRB) that ensures any risks are minimal. While the research team and IRB will do everything possible to minimize risks, it cannot always be guaranteed. It is important to take note of the potential risks you may incur and decide if these possible risks are worth the benefits. Will I be okay with the possibility of not being selected? Clinical trials are designed with certain criteria and researchers are often looking for a select group of participants with certain medical histories. These characteristics and medical histories are outlined in the trial’s inclusion and exclusion criteria. One of the main reasons people are excluded from a study is to maintain their safety. For some clinical trials, you may agree to participate and sign an informed consent document, but then be found to be ineligible for the trial after further testing. If you are not selected for a particular trial this does not mean that you cannot try and enroll for a different clinical trial. There may be other questions you have about clinical trials. You may want to talk with friends, family, and your doctor, or ask the community for their experience. Ultimately, it’s important you make a decision that you feel comfortable with. [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site" title="Sign up for emails from {{site-name}}:"] Clinical trials may be appropriate options for many lung cancer patients. When I had my diagnosis, my oncologist wanted to place me in a clinical trial. Unfortunately, due to the nature of trials, my timing was off -- the trial I would have been best suited for closed before my diagnosis was complete, another would not open for several months and I was eager to begin treatment. After nearly three years, I was considered for another clinical trial -- this time, I did a new panel looking for DNA mutations in my cancer and discovered that I would not qualify for the trial, but the mutations this test revealed included one appropriate for a targeted drug that was relatively new to the market. So in my case, while I had twice hoped for inclusion in clinical trials that seemed promising, neither was deemed appropriate but I still was steered toward effective means of keeping my cancer in check. I have known other patients who have had success with clinical trials. Evaluating the Risks and Benefits Clinical trials offer new hope to many patients, especially those for whom standard therapies have not worked. There are also different types of clinical trials. As a first-line treatment, most patients would be better suited to try established therapies first, or go with a late-phase clinical trial for which effectiveness has already been established. Of course, not every patient would qualify to be a part of every clinical trial. Each trial has its own requirements, which could include having already (or never) tried chemotherapy, exhibiting specific genetic mutations, or being diagnosed at a particular stage. Taking the time to go over available trials with an oncologist is a worthwhile endeavor, regardless, because it will help to expand the dialogue between patient and doctor regarding treatment expectations and opportunities. Certainly, there are some risks involved with clinical trials. After all, the drugs are new and have only had limited testing. But in order to progress to the later phases where patients are given precision doses of these new drugs, the science must have a very solid foundation. Often there is little question about whether the drugs work and more of a question about what the best dose is or what side effects will result. Discussions with a patient's medical team need to outline the broad risk and reward potential. But just as with standard treatments, it must be remembered that every patient is unique and will respond differently. That always includes variables for how effective a treatment is, how long it can be tolerated by the patient, and how long the results will last. Understanding Clinical Trial Phases Clinical trials are performed in phases, with different requirements and objectives, as part of the lengthy approval process required to get a drug to market. Phase 0 A Phase 0 clinical trial is purely exploratory, involves relatively few patients, and often has a very narrow objective. These early human studies may simply allow researchers to know if the drug is even viable to explore further and they may require a lot from the patient participants in terms of further testing. While patients in a Phase 0 trial may not benefit from the treatment, these may be extremely important studies regarding the development of future therapies. However, Phase 0 studies are not required as part of the process and they are only done as researchers feel they are necessary. Phase I Phase I clinical trials are done to establish the safety of a new drug and are usually the first human studies done. This is where side-effects are first monitored. There are no placebos and the doses, at least initially, are generally rather low. These trials are limited to a small number of people and are considered high-risk. Phase II Phase II clinical trials occur after safety has been established. While rare side-effects may still remain undiscovered, the main purpose of Phase II trials is to determine if the drug provides the benefit researchers are looking for. Trials at this phase vary in size, but the patients usually fit very similar profiles (including type and stage of their cancer). No placebos are used in Phase II trials, but different groups may have different doses of a drug to compare which dose works better or is safer. Phase III The critical purpose of a Phase III clinical trial is to determine whether this new drug is actually better -- safer or more effective -- than existing treatments that have already been approved for the marketplace. These are the "randomized" trials that patients often hear about. Participants will not know which treatment they are getting, whether it is the standard drug already on the market or the new drug being tested. These are considered "double-blind" studies when neither the patient nor the doctor knows which treatment the patient is receiving. Placebos are only used if there is no existing standard treatment. Phase III trials are generally quite large, often involving numerous groups around the country or even in multiple countries. Phase IV (Post Marketing Surveillance Trials) After receiving FDA approval, some drugs continue to be studied with Phase IV clinical trials, also known as Post Marketing Surveillance Trials. These long-term studies are designed to answer additional questions about the drugs, usually involving the safety or benefits of extended use, and they may utilize very large groups of patients. In general, the effectiveness of a treatment has been well-established by the time these trials are performed (after all, the drug is already on the market). With the different types of clinical trials available and the wide range of drugs being studied, it is always a good idea for a lung cancer patient to discuss these potential options with an oncologist. In some cases, participation in these studies may help with the development of game-changing treatments that could alter the landscape of cancer care for generations to come. In other cases, patient participants may receive radical results that would not have been possible under standard care. While not appropriate for every patient, and while there are no guarantees that any new therapy will work the way it is expected to, clinical trials remain resources for care worth exploring.1-3 Editor's Note: We are extremely saddened to say that on October 21, 2018, Jeffrey Poehlmann passed away. Jeffrey’s advocacy efforts and writing continue to reach many. He will be deeply missed. [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site" title="Sign up for emails from {{site-name}}:"]

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