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What is a clinical trial?

A clinical trial, also called clinical research, is a research study performed to test the safety and effectiveness of a medical treatment. Study volunteers are closely monitored by medical professionals while receiving an experimental treatment in a controlled environment. Clinical trials are conducted in a careful and thorough manner, and are thus the fastest and safest way to find treatments for humans that work.

What happens during a clinical trial?

A clinical trial studies the effects of a newly developed medical treatment on human volunteers. The study begins with a thorough check of a participant’s health by a professional health care team. Some study participants receive the investigational medical treatment. Other participants do not receive the test treatment, but instead are given the current standard treatment or a placebo as part of a control group. All participants are closely monitored by medical professionals to follow their progress. When the trial is over, the health care team will stay in contact with the participants to follow up on their experience. The study is the most successful when participants follow the protocol carefully and stay in frequent contact with the health care team.

Who can participate in a clinical trial?

All clinical trials have guidelines about who can participate. Guidelines are based on many factors such as age, gender, type and stage of disease, medical history, and previous treatment history. Before participating in a clinical trial, a participant must qualify for the study. It is important to keep in mind that study selection criteria are not used to include or exclude people personally. The criteria are used to identify appropriate participants and keep them safe.

What should I consider before participating in a trial?

You should know as much as possible about the clinical trial before you agree to participate. Taking part in any research study is your choice. It is important that you feel comfortable asking any questions that you may have.

Some questions you may want to ask include:
  • What is the purpose of the study?
  • Who is sponsoring the study?
  • Who has reviewed and approved the study?
  • Who is going to be in the study?
  • Why do researchers believe the new treatment being tested may be effective?
  • Has the treatment been tested before? If so, to what extent?
  • What kinds of tests and treatments are involved?
  • How do the possible risks, side effects, and benefits in the study compare with my current treatment?
  • How might this trial affect my daily life?
  • How long will the trial last?
  • How often do I have to go to the study site?
  • Who will pay for the treatment?
  • Can I talk to other people in the study?
  • What are my responsibilities during the study?
  • Will I be able to take my regular medications during the trial?
  • Who will be in charge of my care?
  • Will the study researchers work with my primary health care physician while I am in the study?
  • How will I know that the treatment is working? Will results of the trials be provided to me?
  • What type of long-term follow up care is part of this study?

What is informed consent?

Before you take part in a study, it is important to fully understand it and to understand what participation may be like. Researchers will help by providing an “informed consent” statement. This is a document that has detailed information about the study, including its length, the number of visits required, and the medical procedures and medications in which you will take part. The document also provides expected outcomes, potential benefits, possible risks, any available treatment alternatives, expenses, terms of confidentiality, and contact information for people you can call if you have questions or concerns. When needed, a translator may be provided.

Researchers will review the informed consent statement with you and answer your questions. If you decide to participate after reviewing the statement, getting all the information you need, and talking with staff and your family, you will need to sign the informed consent statement. Your signature indicates that you understand the study and agree to participate voluntarily. You may still leave a study at any time and for any reason even after signing the informed consent document.

Sometimes, a potential participant may not be able to give informed consent because of memory problems or mental confusion. Someone else, usually a family member with a durable power of attorney, can give consent for that participant. That caregiver must be confident there is small risk to the participant, and that he or she would have agreed to consent if able to do so.

What is an Institutional Review Board?

An institutional review board (IRB), also known as an independent ethics committee or ethical review board, is a committee that has been formally designated to protect the rights and welfare of people involved in research. An IRB often conducts some form of risk-benefit analysis in an attempt to determine whether or not research should be done. The number one priority of IRBs is to protect human subjects from physical or psychological harm.

What are the benefits of participating in a clinical trial?

By participating in a clinical trial, you can take a more active role in your own health care. You may gain access to new research treatments before they are available to the general public. You can also help others by contributing to the development of new medical treatments. Everyone who participates in a clinical trial is playing an important role in medical treatment advancement.

What are the risks of participating in a clinical trial?

The protocol associated with the treatment may require more time and attention for you than if you were receiving a non-protocol treatment. This may involve trips to the study site, more treatments, hospital stays, or complex dosage requirements. It is possible that the medical treatment being studied may not be effective for you. You may also have side effects to the treatment that are unpleasant, serious, or even life-threatening.

What are my rights as a research participant?

As a research participant, you have the right to stop participating in the study at any time. You have the right to ask questions and be given information about the study.

Will my privacy and safety be protected?

Yes. The ethical and legal codes that govern medical practice also apply to clinical trials. Clinical research is regulated by the federal government with strict guidelines and safeguards to protect study participants. Trials follow specific, controlled protocols to help ensure the safety of all participants. All medical records and research material that identify you will be held confidential, as required by law. Only people associated with the study will have access to your personal information. If you are considering volunteering for a clinical trial, the medical practice staff will provide you with informed consent documents that include details about the study. Participating in a clinical trial is an important decision, so volunteers are encouraged to ask questions about the study and consent forms before making a decision.

Can a participant leave a clinical trial after it has begun?

Yes. A participant can leave a clinical trial at any time. Before withdrawing from a trial, the participant should inform the research team and state the reason for leaving the study.

Do I have to have medical insurance to participate in a clinical trial?

No. Medical insurance is not required to participate.

Will I continue to work with my primary health care provider while in a trial?

Yes. Most clinical trials provide short-term treatments for a specific illness or condition. The trial does not provide extended or complete primary health care. By having your primary health care provider work with the research team, you can ensure that your other medications and treatments will not conflict with the study protocol.

What are the phases of clinical trials?

Clinical trials are conducted in phases, with a different purpose for each phase:

  • Phase I trials test a new medical treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. Phase I typically last between one and two weeks.
  • Phase II trials involve giving the study treatment to a larger group of people (100-300) to see if it is effective and to further evaluate its safety. This phase can last from several months to several years.
  • Phase III trials involve giving the study treatment to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely. Phase III can also last from several months to several years.
  • Phase IV trials are studies done after the treatment has been approved for marketing. These studies continue testing the treatment to evaluate its effect in various populations and any side effects associated with long- term use. This final phase can last many years.