• What is a clinical trial?
• What are the phases of a clinical trial?
• What are the pros and cons of participating in a clinical trial?
• What rights and measures protect clinical trial participants?
• Is participation in a clinical trial right for me?
• What questions about clinical trials should I ask my doctor?
• Will I be responsible for any clinical trial costs?
• How do I enroll in a clinical trial?
• Whom can I talk to about participating in a clinical trial?

Q: What is a clinical trial?
A: A clinical trial, commonly referred to as a clinical study or research study, is designed to determine if a new drug or treatment will work on a disease or will potentially be of benefit to patients. Trials allow doctors and researchers to gain information on the benefits, side effects, and possible applications of new drugs, as well as different combinations, doses, and new indications of existing drugs. As a result, the medical community may be able to determine new ways to detect, diagnose, avoid, and control clinical factors responsible for disease.

Q: What are the phases of a clinical trial?
A: Clinical trials have four research phases.

A Phase I study is the first use of a new drug or a new drug indication in humans. It is used to test for safety and side effects, and to determine how the drug should be given. Only a limited number of patients are accepted for participation in Phase I trials.

If the Phase I trial shows safety, a Phase II trial is initiated to investigate the ideal dosing range of the drug. The therapy is tested in a larger number of patients to determine how well it works, and other possible, less common side effects.

In a Phase III trial, experimental treatments are compared to placebo (an inactive substance that looks like the experimental treatment) or common or standard therapies. Patients who participate in Phase III trials are randomized (randomly chosen) to receive the experimental treatment or to receive placebo or a standard therapy. Those patients who are selected to receive placebo or standard therapy are referred to as the control group. The purpose of a Phase III trial is to determine if patients receiving the experimental therapy do better than those in the control group.

A Phase IV clinical trial may be conducted to continue evaluation of the safety and efficacy of therapies that are already FDA approved and available for general use.

Q: What are the pros and cons of participating in a clinical trial?
A: In addition to receiving high-quality care, there are many benefits to enrolling in a clinical trial. Your participation could potentially benefit others by increasing knowledge about a disease and its potential treatments. Also consider that, as a clinical trial participant, you may be one of the first patients to experience positive results from a new therapy. On the other hand, it is possible that new treatments may not provide any benefit and/or you may experience unexpected side effects. Also, you may be placed in a control group that receives placebo (an inactive substance that looks like the experimental treatment) or standard care instead of the study treatment. These are factors you need to be comfortable with before deciding to join a clinical trial.

Q: What rights and measures protect clinical trial participants?
A: Clinical trials are regulated by a number of governing groups and processes to help protect patient safety and the ethicality of scientific research. The rights of clinical trial patients are most notably protected by Institutional Review Boards (IRB), peer review, and the Informed Consent process.

Institutional Review Board (IRB) All research organizations have an Institutional Review Board (IRB) which oversees the actions and documents related to the clinical trial process. This group is made up of medical specialists, nurses, social workers, medical ethicists, and sometimes patient advocates and clergy, who have no special interest in any specific trial. Each member is responsible for ensuring that all trials are conducted safely, fairly, ethically, and with full patient consent and involvement.

Peer Review: Clinical trials are often reviewed by experts such as the National Cancer Institute, or a pharmaceutical company chosen by the study sponsor. Prior to IRB submission, these groups review the trials for scientific merit, patient safety, and ethical considerations. Many research organizations have peer review groups in addition to their IRBs.

Informed Consent: Informed Consent is a process designed to inform you as a clinical trial patient and to help foster good communication between you and the research team. This process includes providing you with information about significant safety issues that might surface during the course of the study. When you decide you might be interested in a particular trial, you will first meet with a member or members of the study research team. They will talk to you about the trial and provide written information about the trial, called the Informed Consent document. This document will relate facts about the trial, such as its purpose, duration, required or alternative procedures, benefits, and risks. After this initial meeting, you may be given some time to read the document and think over your decision. If you decide to join the trial, you will be asked to sign the document, verifying that you understand and agree to its conditions. If you have questions or do not understand any part of the document, do not hesitate to call your contact person. You will want to keep a copy of the Informed Consent document for reference. You may be asked to re-sign the Informed Consent document if any serious events occur.

Once you are enrolled in the trial, your Informed Consent rights continue. You may receive updates of new information which may affect your decision about staying in the trial. To ensure you are still comfortable with participating, your healthcare team will also routinely evaluate how the treatment is affecting you. At all times, you are encouraged to ask questions, and you are entitled to honest, complete answers. Remember that you are free to leave a trial at any time, for any reason.

Q: Is participation in a clinical trial right for me?
A: You will want to explore and discuss any decision about your condition with your doctor, family, and advocacy and support groups. You can gain a better understanding of the benefits and risks of a clinical trial by talking to patients with clinical trial experience, or to those considering participation. Ask your doctor about potential side effects, as well as their length and manageability. If you and your doctor find a trial that you both agree could be beneficial, take the time to understand how participating in the trial might affect you. However, keep in mind that clinical trials require a commitment from both the researchers and the participants. You must be able to put aside some time and energy to receive treatments and undergo monitoring and follow-up. If you cannot fulfill these requirements, you may want to reconsider joining a clinical study at this time. However, even if you join a trial, it is your right to drop out if you decide it is not right for you.

Q: What questions about clinical trials should I ask my doctor?
A: You should feel free to ask your doctor any questions you may have about clinical trials. Before deciding to enter a clinical trial, you should have the answers to the following questions:

• What is the purpose of the study?
• Who is sponsoring the trial?
• What are the qualifications for entry into the trial?
• How many patients will be in the trial?
• What is required of me in the study?
• What are the risks involved during the study?
• Are there risks involved after the study is complete?
• Will I be in any discomfort or pain?
• What kinds of tests, procedures, or treatments will be performed? How many and how often?
• Will there be follow-up after the trial?
• Will I be given other drugs or treatments during the trial?
• What are the chances I will be placed in a control group?
• How will I know if the treatment is working?
• What other options are available for my condition?
• Will I be able to see my own doctor during the trial?
• How will the study affect my daily life?
• What are my options if I do not participate or if I drop out of the study?
• How long will the trial last?
• Whom can I contact if I have questions?

Q: Will I be responsible for any clinical trial costs?
A: In most cases, both the patient and the trial sponsors are financially responsible for clinical trial treatment costs. As a patient, you may be responsible for routine costs, such as doctor visits, hospital fees, and lab tests. Costs normally incurred as part of routine treatment may be covered by health maintenance organizations (HMOs), or by private insurance.

Q: How do I enroll in a clinical trial?
A: You may enter a trial under the guidance of your doctor or if you are referred from another institution. Your doctor may be able to refer you to a specific trial for which you may qualify. If you wish to find out about your clinical trial options independently, there are many resources available on the World Wide Web.

Q: Whom can I talk to about participating in a clinical trial?
A: In addition to discussing this important decision with your family and a supportive circle of friends, you may want to speak with your doctor, nurse, and other clinical trial patients. Advocacy and support groups can give you more information about specific trials and can offer resources to help you make the best decision.