Clinical Trials for Cancer

What are clinical trials?

“The treatments we use today were discovered, tested and first made available to patients in clinical trials—and the drugs that are the future of cancer treatment are in trials today. I want to emphasize that being in a clinical trial is how you get access to the next generation of cancer treatment.” 
David CarboneMD, Ohio State University

Clinical trials for cancer are research studies that compare the most effective known treatment for a specific type or stage of cancer with a new approach. This can be a new drug, or combination of drugs or a different way of using established therapies. There are trials that involve new approaches to surgery and radiation therapy. There are clinical trials for every type of cancer. While many trials focus on late stage disease, there are also trials to prevent cancer, improve early diagnosis, stop the cancer from coming back, reduce side effects or improve quality of life.

Cancer clinical trials are:

  • The engine that drives progress in cancer treatment
  • The only way to find out if a new drug or treatment is better than the existing standard of care
  • In many instances, the only way for a patient to get a new or experimental approach before it is approved for general use
  • A proven way for people facing cancer to get high quality treatment and care
  • A chance to benefit from a new therapy or approach
  • An opportunity to help future cancer patients and contribute to research
  • A way to be involved in the frontlines of research to advance cancer care

Clinical trials for cancer—phases

PHASE I: Once a drug is approved for human studies, it is tested in a small trial to determine the optimal safe dose. Phase I studies often involve patients with different kinds of cancer, or more recently, a single genetic change.

PHASE II: If a drug can be given safely to people on the Phase I trial, it is tested in a Phase II study. These are larger studies, usually for one or more specific types and stages of cancer. The goal of Phase II studies is to both determine the optimal dosing and provide an early assessment of whether the drug works.

PHASE III: These trials take place after a drug has shown good results in earlier studies. They are large studies, often involving hundreds, or even thousands of patients, in multiple centers in the United States and/or abroad. Patients on Phase III trials have specific types and stages of cancer. Many Phase III trials are randomized—meaning that patients are randomly assigned to receive either the new treatment or the established standard of care. These trials are designed to provide definitive evidence to support FDA approval of the drug or agent for use in the public.

PHASE IV: These trials take place after a drug is approved and are often called post-marketing trials. The goal is to make sure that no safety or other concerns come up after a drug is approved that may not have been seen in the pre-approval trials. It is important to follow patients for a number of years to determine if there are any long-term side effects or other issues that affect the way the treatment is used.

Common Questions About Clinical Trials

When should I think about a clinical trial?

“I was diagnosed with a rare and pretty nasty sarcoma. My doctor suggested that I go on a trial to see if chemotherapy before the surgery and radiation helped shrink the tumor and reduce the risk it would come back. I knew that meant I would have more aggressive treatment and more side effects—with no guarantee that it would make any difference. But I also knew the odds if the cancer came back. I chose the trial.”
—Doug, sarcoma patient

A cancer clinical trial may be a treatment option for anyone who has received a cancer diagnosis. The best way to learn more about your specific options is to talk to your doctor, nurse or other members on your health care team. If your doctor is not involved in doing clinical research or does not explain your options for a clinical trial, consider getting a second opinion.

A clinical trial can be particularly important if:

  • You are diagnosed with cancer that has spread or your cancer recurs or comes back after your primary treatment
  • You are interested in getting access to the newest cancer treatments
  • You have a rare or difficult to treat cancer
  • Your treatment that was working has stopped working
  • Your cancer requires that you remain on treatment for a long period of time or the rest of your life
  • You are interested in helping other cancer patients today and tomorrow by advancing cancer treatment
  • Your treatment involves significant side effects and the trial is designed to reduce the impact of these on your life



Information provided by Cancer Support Community.


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