Frequently Asked Questions About Clinical Trials What is a Clinical Trial?A clinical trial (also clinical research) is a research study in human volunteers to answer specific health questions. Before new drugs and other therapies can be marketed, their usefulness, safety and effectiveness must be well-understood. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in children and ways to improve children’s health. Why participate in a clinical trial?Clinical trials offer access to new research treatments before they are widely available. Children and families who participate in clinical trials help others by contributing to medical research and medical knowledge. What happens during a clinical trial?The clinical trial team includes doctors, nurses, and research coordinators, as well as other health care professionals. They check the health of your child at the beginning of the trial, give specific instructions for participating in the trial, and monitor your child carefully during the trial. Some clinical trials involve more tests and doctor visits than your child would normally have for an illness or condition. For all types of trials, you and your child work with a research team. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff. What is a protocol?A protocol is a study plan that is carefully designed to safeguard the health of your child as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages and the length of the study. While in a clinical trial, your child will be seen regularly by the research staff to monitor your child’s health and to determine the safety and effectiveness of treatment. What is informed consent?Informed consent is a process where you and your child receive a thorough explanation about the research study and have the chance to ask questions and discuss participation with your doctor or nurse. As part of this discussion, you will be asked to sign a consent form indicating that you understand the study and are willing to participate. The risks, potential benefits and inconveniences of any tests or procedures are explained in the informed consent document. The document describes your rights as a participant and actions to protect your confidentiality. You will be encouraged to ask questions until you feel comfortable that you understand the study and what is involved should you and your child decide to participate. If your child is under the age of 18, only the child’s parent or legal guardian can sign a consent form. In some cases, children over the age of seven years will be asked to read and sign an Assent form, indicating that he or she agrees to participate. The informed consent process continues throughout the study and you will be encouraged to ask questions at any time. You and your child may withdraw from the trial at any time without any type of penalty. What are the benefits and risks of participating in a clinical trial?Benefits Since the drug or other therapy is still being tested, many clinical trials may not offer direct benefit for your child. However, clinical trials that are well-designed and well-executed help you and your child gain access to new research treatments before they are widely available and provide opportunities to help others in the future by contributing to medical knowledge. Risks There may be risks to your child by being in a clinical trial. There may be unpleasant, serious or even life-threatening side effects to experimental treatment. The experimental treatment may not be effective for your child. The protocol may take more time and attention than would a non-protocol treatment, including trips to the study site, more treatments or complex medication requirements. How is the safety of your child being protected?The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built in safeguards to protect your child. Before any study can be offered at CCMC, it must be approved by a special committee responsible to protect human subjects in research, known as the Connecticut Children’s Medical Center Institutional Review Board (IRB). The IRB is an independent committee of physicians, community advocates and others who assure that a clinical trial is ethical and the rights of your child are protected. The IRB must review, approve and monitor all research at Connecticut Children’s Medical Center. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. What you should consider before allowing your child to participate in a trial?You and your child should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for you to discuss with the health care team. Some of the answers to these questions are found in the informed consent document. What is the purpose of the study? Who is going to be in the study? Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before? What kinds of tests and experimental treatments are involved? How do the possible risks, side effects, and benefits in the study compare with my child’s current treatment? How might this trial affect my child’s daily life? How long will the trial last? Will hospitalization be required? Who will pay for the experimental treatment? Will I be reimbursed for other expenses? What type of long-term follow up care is part of this study? How will I know that the experimental treatment is working? Will results of the trials be provided to me? Who will be in charge of my child’s care? Can my child leave a clinical trial after it has started?Yes. Your continued participation is voluntary. You and your child can leave a clinical trial, at any time. When withdrawing from the trial, you should let the research team know about it, and the reasons for leaving the trial. Where do the ideas for trials come from?Ideas for clinical trials can come from laboratory research or from clinical practice. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work. Who sponsors clinical trials?Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran’s Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors’ offices, or community clinics. As a Research Subject you and your child have a right to information about: Why the study is being done What will happen during the study How participating would change your child’s medical care The discomforts and risks of the things that will be done to your child in the study Possible benefits of taking part in the study Other treatments that are available if your child chooses not to be in the study Medical treatment if problems occur during the study You and your child also have the right to: Decide NOT to take part in the study Not be pressured to take part in the study Stop being in the study at any time Ask questions before, during, and after the study Receive information throughout the study What is clinical research?Clinical research allows us to be at the cutting edge of the latest clinical treatments and practices. Sometimes being asked to participate in a clinical research study can be confusing. We hope this information will help you understand more about research and allow you to decide if you would want your child to be in a research study if asked to participate.