Pediatric Cancer Services

Clinical trials, protocols and research

Much of the dramatic progress made in treating childhood cancers in recent years is because of advances made through research. Less than 1 percent of adult cancer patients participate in clinical trials or research studies of cancer treatment; with children, that number is 60 percent. Thanks to this approach, treatments for childhood cancers have been refined and highly specialized for each type of cancer. New treatments are tested scientifically in clinical trials.

A child with cancer may be offered the chance to participate in a protocol. Protocols are treatment plans that several hospitals use for treatment of a particular type of cancer. Protocols are also a way to standardize care of children with cancer. Protocols, or clinical trials, are carefully designed by a panel of world experts in a particular type of childhood cancer. Even though the treatments given in clinical trials are considered new or “investigational,” there is wide agreement among pediatric cancer specialists that they are the best known currently.

The Children's Oncology Group (COG), a national organization, is responsible for planning, conducting and evaluating these clinical trials. Most of the protocols used at Yale-New Haven Children's Hospital are developed through COG. The pediatric hematology/oncology department of the Yale University School of Medicine is a full participant in COG. This means the treatments offered are at the leading edge of therapy for childhood cancer and are identical to what would be offered at numerous other children's hospitals throughout the country.

Before a trial can begin, it must be approved by the Yale-New Haven Institutional Review Board (IRB), which is composed of physicians. IRB approval means the trial has met the board's standards for safety, scientific progress and purpose.

Phases of clinical trials

As a new drug is developed, it moves through a sequence of clinical trials. Cancer clinical trials are divided into three phases.

Phase I trials usually involve only a small number of patients and attempt to determine safety and the best dosage of the drug.

Phase II trials investigate whether certain types of cancers respond to a drug. Generally more patients are enrolled in these trials. Combinations of drugs may also be evaluated in Phase II.

If the therapeutic agents appear promising, Phase III trials compare the results from the experimental treatment to standard therapies already in use. Researchers may ask whether the new drug is better than the current treatment and how side effects compare. Phase III trials usually involve hundreds of patients and are performed at the same time at many children's hospitals.

Yale Cancer Center clinical trials

Yale Center for Clinical Investigation

Last revised: Feb. 20, 2008 (jy)

All information is intended for your general knowledge and is not a substitute for medical advice or treatment for specific medical conditions. You should seek prompt medical care for any specific health issues and consult your physician before starting a new fitness regimen.