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What are cancer trials?

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Health care providers, learn more about cancer clinical trials here.
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Key facts about cancer trials

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Cancer clinical trials, also called cancer trials, help advance medical research that could benefit people living with cancer now and in the future. A clinical trial is a voluntary research study that is conducted to help answer important, specific questions about the safety and effectiveness of:

  • Investigational treatments or therapies
  • Different ways of using existing treatments
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There are many types of cancers and many different cancer clinical trials in the United States alone. One key difference between cancer clinical trials and most other types of clinical trials is that cancer clinical trials rarely use placebos or no treatment as one of the treatment groups. In fact, those situations should only occur when the standard treatment is to watch and wait.
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People are living longer today because of the outcomes of past trials. 1 Cancer studies are critical in helping to test and develop new research treatments and these emerging treatments may help extend the life of a person living 
with cancer.
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Find a cancer trial near you

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Search for cancer clinical trials in the United States.
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Discover a trial for you
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https://leal.health/clinicaltrials
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How cancer trials work
How cancer trials work
Different types of cancer trials
Different types of cancer trials
Phases of cancer trials
Phases of cancer trials
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How cancer trials work

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It’s important to consider all of your options when you or a loved one are diagnosed with cancer, including participating in a cancer clinical trial. You or a loved one will know what to expect before taking part in a study—and the research team is committed to open and caring communication throughout a study.

Participating in a cancer clinical trial gives you the opportunity to access emerging research treatments while being cared for by doctors and nurses connected with the study.

In most cases, when you enroll in a clinical study, you will receive either a currently approved standard treatment or a new study treatment that is undergoing multiple phases of testing and review to see if it is safe and effective. It is worth nothing that placebos (medicines with no active ingredients) are rarely used in cancer trials, and typically only when there is no current treatment or when it is being compared to a current treatment.

Taking part in a clinical trial is your choice. If you decide to take part, you may leave at any time for any reason.
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How cancer trials work
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Different types of cancer trials

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Clinical trials may help diagnose, treat, and prevent cancer. There are different types of clinical 
trials, including:
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Treatment trials
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These trials test investigational treatments. They might test potential new medicines, devices, or different drug combinations. A trial could also help develop a new approach to radiation therapy or surgery. For people living with cancer, this is the most common type of trial.1
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Other types of trials
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  • Prevention trials
  • Screening trials
  • Diagnostic trials
  • Supportive care/quality-of-life trials
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In addition to the main types of cancer studies, there are also four important phases of a research study.
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Different types of cancer trials
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How new therapies become approved treatments
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Before a new medicine can be approved by the U.S. Food and Drug Administration (FDA), it must be tested in different sets of studies called "phases." Knowing what phase a trial is in can help answer important questions that you or a loved one may have.
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Cancer trials phases

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Phase 1—about 20 to 100 participants
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In a Phase 1 clinical trial, the research treatment is tested on a small group of people to determine the best dose of the research treatment as well as discover the most common side effects.

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Phase 2—up to a few hundred participants
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In a Phase 2 clinical trial, the research treatment will be given to a larger group of people to investigate how well it works, collect more information on safety, and determine whether the treatment can move into a Phase 3 clinical trial.
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Phase 3—about 300 to 3,000 participants, depending on how common the type of 
cancer is
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The goal in this phase is to see how safe the research treatment is and how well it works compared to a standard approved treatment. The number of participants will depend on how common or rare the type of disease or condition being studied is.

In a Phase 3 cancer study, participants will be assigned to either a group that receives a standard treatment or a group that gets a research treatment. Participants are randomly assigned, which means they won’t know which group they will be placed in, but they have an equal chance to receive either treatment.

The outcomes of the two groups are compared to see which group benefitted more. If the group that received the research treatment benefits more than the one that received the existing approved treatment, the research data is typically used to file for FDA approval. This is done so that the research treatment can be approved for doctors to start prescribing the treatment.

Why Phase 3 Is So Important

Out of all the trial phases, Phase 3 cancer clinical trials usually include the largest number of participants. This allows the participants to be diverse and reflective of the people who are most commonly diagnosed with the type of cancer being treated in the trial. The results of a Phase 3 trial are pivotal as they provide comprehensive safety and efficacy information.

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Phase 4—several thousand participants
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In this phase, trials are conducted to examine the medication after it has been approved by the FDA and is available for doctors to prescribe. A Phase 4 cancer study looks at the real-world outcomes of a drug treatment, including the safety of a drug over time, how well it works, and a patient's quality of life while on the medication.

If a cancer trial hasn't been brought up by your doctor, it doesn't mean that you’re not eligible. You should always ask your doctor to find out if there are ongoing trials that might benefit you.
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See cancer trial benefits & risks
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/science/clinical-trials/cancer/potential-benefits
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Phases of cancer trials
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Find a cancer trial near you

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Search for cancer clinical trials in the United States.
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Discover a trial for you
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References

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1 Unger JM, LeBlanc M, George S, et al. Population, Clinical, and Scientific Impact of National Cancer Institute's National Clinical Trials Network Treatment Studies. J Clin Oncol. 2022;41(11):2020–2028.
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