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Common Types

What is a Clinical Trial?  

Clinical trials are studies of new drugs or procedures that involve people, as opposed to trials involving animals or studies done in the lab. Through clinical trials, doctors learn whether a new treatment is safe and effective for patients. Such trials are critical to the development of new treatments for cancer.

For a child facing cancer, and for their family, choosing whether or not to take part in a clinical trial can be a difficult decision. While clinical trials can yield great advances, they also offer no guarantee, so  it is important for children and families to learn everything they can before committing to a trial, through a process called Informed Consent.

Why Participate in a Clinical Trial?

Every advancement in curing childhood cancer can be credited to research, and clinical trials are central to research efforts.

There is a definite connection between supporting clinical trials and improving survival rates. Relatively few adults participate in clinical trials, but most children do take part. Meanwhile, the survival rates for pediatric cancer have increased significantly in the past 20 years, while rates for adult cancers have not. 

Participation is paying off. In fact, since the 1950s, survival rates for childhood cancer have soared: from less than ten percent to more than  77 percent. And while cure rates vary according to each specific type of childhood cancer, finding cures for any form truly does depend on clinical trials and research.2

Informed Consent

Informed Consent is the process by which potential study participants and their families learn key facts about a clinical trial before deciding whether or not to participate. The potential study participants and their families talk with the research doctor or nurse about the trial and review a written consent form. The consent form includes details about the study approach, the intervention given in the trial and the possible risks and benefits and tests associated with the trial.

Most children with cancer enroll in clinical trials, and most clinical trials for childhood cancer are conducted by the National Cancer Institute's sponsored cancer cooperative Children's Oncology Group, a merger between the Pediatric Oncology Group, Children's Cancer Group, Wilms Tumor Study Group and Intergroup Rhabdomyosarcoma Study Group.3

The other main sponsors of clinical trials are pharmaceutical and biotechnology companies, which must show that their medicines are safe and effective before they can be marketed. But in many cases, pharmaceutical and biotechnology companies do not sponsor research for rarer diseases, such as childhood cancers, because they feel that those investments will not bring about a large enough return.

There is no right or wrong choice when it comes time to decide on taking part in a clinical trial. The decision is personal. Each patient must weigh the benefits and risks and consider what he or she hopes to achieve by taking part.

Learn about cancer research and public policy »

Find out about the disparities surrounding cancer healthcare »

Learn about survivorship challenges »

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Color-coded vials lined up on a tray
Three phases of clinical trials:

Phase I trials evaluate how a new drug or treatment regimen should be administered, how often, and in what dosage. New drugs must show clear evidence of great promise in the lab before being tested on people.

Phase II trials assess efficiency of treatment for particular types of cancer and provide more information about safety.

Phase III trials compare promising new treatments against the best standard treatment to date. If the new treatment proves effective, it may become the new standard.