NCCN Guidelines for Patients® | Myelodysplastic Syndromes

38 NCCN Guidelines for Patients ® : Myelodysplastic Syndromes, 2018 4 Cancer treatments Clinical trials Clinical trials New tests and treatments aren’t offered to the public as soon as they’re made. They need to be studied first. New uses of tests and treatments also need to be studied. A clinical trial is a type of research that studies a test or treatment. Clinical trials study how safe and helpful tests and treatments are. When found to be safe and helpful, they may become tomorrow’s standard of care. Because of clinical trials, the tests and treatments in this book are now widely used to help people with MDS. Tests and treatments go through a series of clinical trials to make sure they’re safe and work. Without clinical trials, there’s no way to know if a test or treatment is safe or helpful. Clinical trials are done in a series of steps, called phases. The four phases of clinical trials are described next using the example of a new drug treatment: † † Phase I trials aim to find the best dose and way to give a new drug with the fewest side effects. If a drug is found to be safe, it will be studied in a phase II trial. † † Phase II trials assess if a drug works for a specific type of cancer. They are done in larger groups of patients with the same type of cancer. † † Phase III trials compare a new drug to the standard treatment or to a placebo—a substance with no effects. These are randomized, meaning patients are put in a treatment group by chance. Phase III trials are done to learn about short- and long-term side effects and about safety. † † Phase IV trials test new drugs approved by the FDA (U.S. F ood and D rug A dministration) to learn more about short-term side effects, long-term side effects, and safety. They involve many patients with different types of cancer. Joining a clinical trial has benefits. First, you’ll have access to the most current cancer care. Second, you will receive the best management of care. Third, the results of your treatment—both good and bad—will be carefully tracked. Fourth, you may help other patients with cancer. Clinical trials have risks, too. Like any test or treatment, there may be side effects. Also, new tests or treatments may not help. Another downside may be that paperwork or more trips to the hospital may be needed. To join a clinical trial, you must meet the conditions of the study. Patients in a clinical trial often have a similar cancer type and general health. This is to know that any improvement is because of the treatment and not because of differences between patients. You also must review and sign a paper called an informed consent form to join a clinical trial. This form describes the study in detail, including the risks and benefits. Ask your treatment team if there is an open clinical trial that you can join. There may be clinical trials where you’re getting treatment or at other treatment centers nearby.