What is a Clinical Trial?

Clinical trials are a care option that you should discuss with your health care team. A clinical trial is research that studies better ways to prevent, screen for, diagnose and/or treat a disease. Almost every cancer treatment that patients receive today, are as a result of a clinical trial and patients who were willing to help science find new and better ways to treat a disease. We’ve curated frequently asked questions to help you explore and better understand your options. You can also use the questions below under “Questions to Ask Your Health Care Team” to help guide discussions with your health care team.

Carlos Arteaga, MD
University of Texas Southwestern
Stand Up To Cancer, Scientific Advisory Committee

What kind of clinical trials are available?

Clinical trials are research studies that compare the standard of care, which are the most effective known treatment available, with a new approach. A clinical trial can exist for all types and stages of cancer from prevention through long-term survivorship.

There are many kinds of clinical trials and a few examples include:

  1. Testing new ways to prevent cancer or recurrence of cancer, which may include testing medicines, vaccines, vitamins and/or lifestyle changes
  2. Testing new medications or new combinations of medication. Medications that work in one type of cancer may also be studied in another type of cancer
  3. Diagnostic trials which study new ways to understand an individual person’s disease
  4. Screening trials that test the best ways to detect the cancer
  5. Quality-of-life trials that explore ways to improve the comfort of people with cancer
  6. Testing of new approaches to surgery, radiation therapy and/or complementary/alternative medicines

How do clinical trials work?

To ensure a clinical trial is safe and effective, researchers conduct clinical trials in different phases. Each phase is designed to answer a separate question.

Phase I trials evaluate safety. Phase II trials measure effectiveness. Phase III trials compare the new treatment to current standard of care. Phase IV trials are conducted to further evaluate new uses or long-term effects of the treatment.

How do I know if a clinical trial is right for me?

Ask, Learn, and Evaluate Your Options.

Before signing up for a clinical trial, it’s important you understand what treatment is offered and how this differs from the standard treatment available for your cancer. Find out about any side effects of the study. Talk to a social worker for emotional support. Ask questions that can help you determine which option is best for you and your lifestyle.

Confirm That You Are Eligible.

All clinical trials have guidelines on who is able to participate in the study, called eligibility criteria. These factors may include age, gender, type and stage of cancer, treatment history, and/or any other conditions you may have.

What are the potential benefits of participating in a trial?


  1. You may have the opportunity to receive new medication, procedures and/or treatments that are more effective or better tolerated than those currently available as a standard of care
  2. You will be helping future generations and making a valuable contribution to cancer research
  3. You will be closely monitored while on a clinical trial by your doctor and research team

What are the potential risks of participating in a clinical trial?


  1. A clinical trial may require more time and medical attention than standard of care. This may include more doctor visits, phone calls, hospital stays, and/or a more complex treatment regimen
  2. The treatment may not work (even if it helps other patients on the clinical trial)
  3. The treatment may cause side effects

What are my rights and protections?

Patients who enroll in clinical trials have rights and protections to ensure their privacy and well-being are maintained. One of the most important protections is called informed consent, which ensures you understand all aspects of the clinical trial, any potential risks and benefits, as well as any financial obligations. You will also be asked to sign a HIPAA form, which allows doctors to use your health information as part of the study report, without sharing your name or other personal details.

Clinical Trials Are Completely Voluntary.

You always have the right to drop out of a clinical trial at any time. If you decide to leave a clinical trial, talk to your doctor. They can provide you with information on how quitting the study may affect your health, and help you evaluate other treatment options available.

What costs are covered?

Although the study drug is often covered, your insurance company may not cover doctor visits, testing, and/or other expenses associated with a clinical trial (e.g. transportation to and from treatment). Make sure you understand what’s covered and any out-of-pocket costs, and how this may differ from other treatment options available to you. Ask about any programs that may be available to help cover your financial obligations and out-of-pocket costs.

What is the difference between standard therapy and investigational therapy in a clinical trial?

Standard therapy is the treatment that experts consider the most effective known treatment available. Health care professionals are obligated to provide patients with standard therapy. Standard therapy is also called standard of care or best practice. In cancer clinical trials, investigational therapy refers to a drug (including a new drug, new dose, combination with other drugs, or route of administration) or procedure that has been tested in a laboratory and received approval from the FDA to be tested in humans. A drug or procedure may be approved by the FDA for use in one disease or condition, but may be considered experimental in other diseases or conditions.

How do I find a clinical trial?

There are a few ways to learn about clinical trials. Ask your oncologist since they are familiar with your case and may be aware of clinical trials you are eligible for. You can also find listings of clinical trials through various resources including:

  1. National Cancer Institute’s Cancer Information Service 1-800-4-CANCER or www.cancer.gov/clinicaltrials
  2. American Cancer Society’s Clinical Trials Matching Service 1-800-303-5691 or www.cancer.org/clinicaltrials

What is a randomized clinical trial?

A randomized clinical trial is a study in which the participants are assigned by chance to separate groups to compare different cancer treatments. Neither the researchers nor the participants choose which group the patient is entered into. Patients are assigned by chance to either a standard treatment group (control group) or investigational treatment arm. In cancer clinical trials, the control group invariably receives the standard treatment currently available. Randomization in a cancer clinical trial gives each cancer patient an equal chance of being assigned to any of the groups. It is one method used to prevent bias in cancer research.

What is a placebo?

A placebo is an inactive substance or treatment that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.

Placebos are rarely used in cancer clinical trials and are never used in place of treatment. Patients who join cancer clinical trials are either given the standard of care, (best treatment available for their specific cancer) or receive a new treatment being investigated. Remember that although you are the only one who can decide whether to take part in a cancer clinical trial, your doctor and the rest of the healthcare team are important and valuable sources of balanced information about the risks and benefits of participation

Are clinical trials just for advanced cancer?

No. Cancer clinical trials exist for all types and stages of cancer, as well as for cancer prevention.

Questions to Ask Your Health Care Team

If you and your health care team have decided that a clinical trial may be an option for you, be sure to ask questions to ensure you fully understand both the risks and benefits, as well as potential next steps. Here are a few questions to get you started:

  1. What clinical trials am I eligible for?
  2. Why is this clinical trial a good fit for me? What are the benefits?
  3. What phase is this clinical trial and what does that mean?
  4. What are the possible risks? Side effects?
  5. What are my treatment options if I don’t join this trial?
  6. How do the possible risks, side effects, and benefits in the trial compare with my current treatment or the standard of care?
  7. Who will monitor my care and safety?
  8. Who will review the information collected during the cancer clinical trial? What type of data/information will I receive?
  9. What kind of tests and treatments are involved in the trial? After the trial? Where do these tests and treatments take place?
  10. Will I be able to see my own doctor? Who will be in charge of my care?
  11. What is the expected timeline for the trial? When will the trial begin? How many visits per week/month will I need to make?
  12. Who will pay for the treatment? Will the trial or my insurance cover all or part of it? What costs am I responsible for? Will I be reimbursed for any expenses such as transportation?
  13. Can I take the informed consent form home to talk it over with my family or care/support partner? If I have more questions about the trial, whom can I contact?
  14. Where can I learn more about this clinical trial?
  15. What support will be available for me and my care/support partners during the trial? Can I talk to other people in the trial?
  16. What is the long-term follow-up care?

Looking for Additional Resources?

We’ve curated additional resources to help you access important information about clinical trials. You can access general cancer information, as well as information that is specific to the cancer you are interested in.

Go to Prescription to Learn®