Understand the basics of clinical trials

TheBasics Clinical trials are research studies that evaluate the safety and effectiveness of new drugs or medical devices. After researchers test new therapies in the laboratory, the most promising experimental treatments are moved into human clinical trials.

Studies, regardless of the outcome, are necessary to advance medical research.

Why would I want to participate in a clinical trial?

People participate in studies for various reasons. You may have an illness and want to explore research and what investigational options may be available. You may be a healthy volunteer who wants to help others and contribute to scientific discovery. The bottom line: Clinical trials may help researchers develop new treatment plans. Regardless of your reason for enrolling, you will be providing hope to future generations.

Who runs a clinical trial?

A principal investigator (PI), is the individual who actually conducts and is responsible for the research / clinical investigation. This person can be a medical doctor or other specialty. Other research team members also monitor your participation to test the safety and effectiveness of the product.

Who sponsors a clinical trial?

Funding comes from a variety of organizations and individuals from both the private and public sector. Sponsors may include physicians, foundations, medical institutions, philanthropic not-for-profits, pharmaceutical companies and government agencies.

How is a clinical trial set up?

Clinical research follows a plan called protocol, which is designed to answer specific research questions. The protocol details how a clinical trial is conducted, who is eligible to participate, the duration of a study and what data will be gathered.

Who approves a clinical trial?

An Institutional Review Board (IRB) approves the clinical trials in the U.S. An IRB is an independent committee consisting of doctors, scientists and members of the community at large whose charter is to safeguard the rights of all participants and hold trials to ethical standards. 

What are the different phases of clinical trials?

Human testing typically takes place over a series of phases, each with a different purpose. Typically, you will be involved in only one phase of a study. While treatments move through phases, people do not.

Phase 0 trials are exploratory studies involving limited human exposure to the drug, without therapeutic or diagnostic goals.

Phase I trials assess the safety of a drug or device given to a small group of people for the first time. Researchers examine how a drug is absorbed and metabolized, and evaluate potential side effects as dosage levels increase.

Phase II trials test the effectiveness of a drug or device. This phase typically lasts up to two years and may involve more than 100 patients. About one-third of experimental drugs successfully complete Phase I and II.

Most Phase II studies are randomized trials in which one group receives the experimental drug, while a second set, called the control group, receives a placebo, or an inactive drug. These trials are often designed as single- or double-blinded studies. In a single-blinded study, patients don’t know if they’ve received the experimental drug or placebo. In a double-blinded study, neither the patients nor the researchers know who has received the experimental drug.

Phase III trials confirm the safety and effectiveness of a drug or device. Researchers often follow a few thousand patients over the span of several years, giving various dosages to different populations. Once a drug or device successfully completes Phase III, it goes to the Food and Drug Administration to assess whether it is approved.

Phase IV trials evaluate long-term benefits and side effects after the FDA has approved a drug or device for the public. Depending on the results, the FDA may remove a drug or device from the market, or restrict its use.

What questions should I ask before enrolling?

If you are thinking about participating in a clinical trial, compile a list of questions. Don’t be shy about asking as many questions as needed in order to feel comfortable. Consider bringing someone with you to help ask questions and record answers. Here are some suggestions to get your started:

About the clinical trial

  • What is the purpose of the study?
  • What are the chances this study will help me?
  • What risks are involved?
  • What time commitment is required?
  • What will my responsibilities be?
  • Can I talk with other people in the study?
  • What happens if I decide to quit?
  • Can the principal investigator remove me from the study if I want to continue?
  • Does this study involve a placebo?

About your care

  • What kinds of therapies or tests will be done?
  • Will I be in pain? If so, for how long?
  • How will the therapies in the trial compare to those I would have outside the study?
  • Who will collect and review my information?
  • Will I still see my doctor during the study?
  • Will I take my regular medications during the study?
  • If I have side effects, can they be treated?
  • What expenses will I incur? Will insurance cover these costs?
  • What are my options besides this study?

How do I sign up?

Before you decide to enroll in a clinical trial, the research team will give you an informed consent document, which lays out the details of the study such as the purpose, duration, risks and benefits, required procedures and tests, and contact information. If you choose to participate, you will sign the informed consent document but may withdraw from the study at any time.

If you have questions about clinical trials or want help identifying a trial in which you may be eligible, call our Patient Engagement Center at 844-552-2734. One of our team members will walk you through the process and connect you with the appropriate researcher and research team.