Clinical Trials 101 – Your Guide to Cystic Fibrosis & 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. Learn more about clinical trials [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site"] Thousands of clinical trials occur each year. Learning about clinical trials can feel like you are learning a new language. The good news is that there are lots of resources to help support and guide decision-making before enrolling in a clinical trial. Check out 10 things to know about clinical trials and participating in trials. Clinical trials are the backbone of treatment breakthroughs Clinical trials allow researchers to understand the impact of a treatment. When it comes to treatments or drugs, clinical trials provide critical information about the drug’s safety and effectiveness. In addition, they can lead researchers down of path to develop other new treatments. Ultimately, clinical trials make a difference not just for today’s patients, but also potentially to those who’ll be diagnosed in the future. Regulatory and ethics groups oversee clinical trials All places that conduct clinical trials have strict ethical duties to participants and regulations they must adhere to. In order to ensure that the ethical and regulatory guidelines are followed, there is a team of people who are independent of the research team who are asked to review and oversee the clinical trial. This team of people is known as an Institutional Review Board (IRB), sometimes known as the Independent Ethics Committee or Ethics Review Boards. The IRB must review the research protocol before any participants can enroll in a study. Risks and benefits are different for each person Each clinical trial has different risks and benefits that are explained in the study protocol. Since each participant is different it can mean that each person may experience the risks and benefits differently. For example, it could be possible that one participant may experience a side effect that another participant does not experience. The exact risks and benefits can be difficult to predict since a component of some clinical trials is exploring the impact of a treatment. During the Informed Consent Process, a member of the research team will review the risks and benefits of the clinical trial. There are several key players involved There can be lots of different people involved in clinical trials, but there are key players to know about. Key players include the Principal Investigator (PI), who is the lead researcher leading the clinical trial. There is the trial sponsor, who is paying for research to take place. The research staff, which can include doctors, nurses, social workers or clinical research staff who will work directly with patients. Lastly, patients and their caregivers, without these key players medical advances wouldn’t be possible! Participants can leave a trial at anytime If you enroll in a clinical trial and then find that you are no longer able to participate, you are allowed to drop out of the trial at any stage. Trial participants do not have to provide any reason for leaving if they do not want to. If a participant decides that they no longer wish to partake in the clinical trial it is the research team’s responsibility to remove the person from the trial in a way that is safe for the participant. The health and safety of participants is the top priority One of the primary responsibilities of the research team is the care and safety of participants who have volunteered for the trial. Researchers may go by unfamiliar titles, like Principal Investigator or Study Coordinator, but they are, also, doctors, nurses, and trained research professionals. As such, they monitor participant safety and well-being. They will adjust study-related care when they feel it’s in the participant’s best interest. This may mean withdrawing a participant from a clinical trial to prevent harm. There are rules about who can and cannot participate The factors that allow an individual to participate in a clinical trial are called the inclusion criteria. The factors that disqualify an individual from participating in a clinical trial are called exclusion criteria. Inclusion and exclusion criteria are typically based on factors such as age, gender, health status, previous treatment history, and other medical conditions. There is a lot of thought and time put into the development of inclusion/exclusion criteria as these factors may have an impact on the findings of the clinical trials. The inclusion and exclusion criteria are applied to all possible study participants equally before they enroll in the study. Clinical trials occur in various settings Clinical trials could take place at a hospital, a university, a community clinic, or a doctor’s office. In addition to a variety of sites, there are a variety of researchers. Some medical professionals devote their career to conducting clinical trial research, while other research professionals conduct research for some of the time and the remaining time they are providing patient care in a healthcare setting. Evaluating the safety and efficacy of new drugs is a multi-step process When you peek inside your medicine cabinet do you ever wonder the journey that a medication underwent in order to allow it to prescribed by a doctor and land itself in your cabinet? It’s a long journey and thorough one. Every potential treatment undergoes multiple phases of research, each with a different goal. In clinical trials that are studying an interventional drug, treatment, or therapy, there is pre-clinical research that occurs in laboratories, usually with cells or animals. If the pre-clinical studies show promising results, the treatment team must get permission from the US Food and Drug Administration (FDA) to test in humans. The research team submits an Investigational New Drug (IND) application to the FDA and must be approved before any next steps. People have lots of different reasons why they participate There are numerous reasons to participate in a clinical trial. Some might decide to participate to gain access to new or alternative treatments. Others may join to have access to personalized and expert medical attention. Some may participate as it can provide access to medications and procedures at no cost to the participant. Some people are motivated to participate to contribute to advancing medicine. Choosing to participate in a clinical trial is an individual decision and each person may have their own personal reasons for deciding to take part in a clinical trial. Join the Cystic-Fibrosis.com community [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site"] When many people think of clinical trials, they may think of testing a new experimental drug or taking a placebo pill. But, not all clinical trials involve treatment! Even among clinical trials that focus on treatment, many don’t involve medication. Researchers conduct clinical trials to study and improve many aspects of healthcare. Here are some of the most common types of clinical trials. Treatment trials Treatment trials may involve a variety of different types of treatment, including medication, a combination of medications, or a new surgery or procedure. While some treatment trials compare a group of people receiving a medication to a group who is receiving a placebo (sometimes known as a sugar pill or an inactive medication), that is not always the case! Many clinical trials are being conducted to find new treatments for people with serious illnesses, like cystic fibrosis. In some situations, it would be unethical to not provide any type of treatment to a trial participant. Often, trials will compare a group of people taking the current best available treatment option, sometimes called the 'gold standard,' to a new experimental treatment. In other situations, researchers may be conducting a clinical trial to test new dosages for an already approved treatment. People in these trials would not receive a placebo medication and may even receive a medication that is available to the public (often for free!). Prevention trials These trials look for ways to prevent disease in healthy people or look for ways to prevent a disease from coming back. Some prevention trials focus on non-medical ways to prevent disease. For example, a trial where participants make a certain lifestyle change could be studying prevention efforts. Other prevention trials may use new drugs or therapies to try and prevent disease. For example, a vaccine trial to prevent an infectious disease. Screening trials Clinical trials focusing on screening aim to find the best ways to detect health conditions or disease. It’s important to note that screening is different than diagnosis. Screening looks to detect a disease in someone before they have developed any symptoms, while a diagnosis confirms a disease after symptoms have presented. Screening trials may include different imaging tests, such as MRI or CT scans, or laboratory tests, like blood or urine tests. These trials may also look at genetics to better understand what role inherited genes play in disease development. Diagnostic trials Diagnostic trials study or compare different tests or procedures that are used to diagnose a disease or health condition. For some conditions, there is no single test that is able to confirm a diagnosis. Many people will need to have several different tests to rule out other conditions before being diagnosed. Diagnostic clinical trials can hopefully make diagnosis an easier process for future patients. Quality of life trials Quality of life trials, sometimes known as supportive care trials, do not focus on curing a disease or treating a health condition. Instead, these trials focus on improving the comfort or quality of life of people with different health conditions. For many people living with conditions such as cystic fibrosis, symptoms and treatment side effects can have a negative impact on day-to-day life and overall well being. These trials recognize that while new treatment breakthroughs are important, overall quality of life is also extremely important to patients. Behavioral trials Trials that focus on behavior aim to compare or evaluate different ways behavior can impact health. While many behavior trials may also focus on prevention, like those looking at diet or exercise, others may aim to better understand how our brains work to shape behaviors. 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