What are Phase 1 Clinical Trials and When are They Done?

When are Phase 1 Clinical Studies Done for Cancer?

male doctor doing research in a lab
What are phase 1 clinical trials?. National Cancer Institute, Rhoda Baer (photographer)

Definition: Phase 1 Clinical Trials

Phase 1 clinical trials are done to see if an experimental medication or treatment is safe. After a treatment is tested in the lab or on animals, it enters a phase 1 clinical trial that is done with humans. These trials usually involve only a small number of people to determine if a drug or treatment is safe and to determine the best dose of a drug and how it should be given (whether orally or intravenously).

Though the primary purpose of these trials is to evaluate safety, they may also determine if a treatment appears to work for a cancer.

If a treatment appears safe at the end of a phase 1 clinical trial, it may then enter a phase 2 clinical trial, a study done to see if a treatment is effective. If a drug or treatment is deemed safe in a phase 1 trial and effective in a phase 2 trial, it will then enter a phase 3 clinical trial. Phase 3 clinical trials are much larger and are done to see if a treatment is not only safe and effective but works better or has fewer side effects than treatments currently available.

Why Would I Want to Be in a Phase 1 Clinical Trial?

There are a few reasons someone may consider participating in a phase 1 clinical trial. One is the hope of advancing research that may help others with your disease in the future. Another is the hope that a new drug or procedure that has not yet been tested on humans will offer a chance of survival when other treatments have failed.

The only way that advances in cancer treatment, and subsequent survival, are made, is via patient participation in clinical trials.  That said, clinical trials are not for everyone. The articles below may help you decide if a trial is right for you, and can direct you to nurse specialists (a free service) who can help you if you are uncertain.

An exciting example of a new treatment first tested in a phase 1 trial and then rapidly becoming the standard of care has recently been seen with ALK-positive lung cancer.

Also Known As: phase I clinical trials

An Example of Phase 1 Clinical Trials:

Ben decided to take part in a phase 1 trial for his lung cancer. The study is designed to see if a new drug is safe. Chances are it will not benefit him significantly, but lung caner runs in his family and he hopes to further research for his children and grandchildren. He also carries a small but real hope that the drug will increase his own survival, after all, all drugs that increase survival were once tested in these trials.

More About Clinical Trials


US Food and Drug Administration. The FDA's Drug Review Process: Ensuring Drugs are Safe and Effective. Updated 11/0614. http://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htm