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Clinical Trials

Originally published on November 10, 2009
Most recently updated on April 10, 2013

Clinical Trials/Protocols

Your child’s doctor or treatment team may recommend that you enroll your child in a clinical trial. This is a research study of new therapies (or experimental drugs and treatments). By studying a larger collected group of children in a protocol with very exact treatment guidelines, doctors are able to draw better conclusions about how effective a treatment is and work to improve it.

The National Cancer Institute (NCI) oversees a large cooperative group of over 240 hospitals—the Children’s Oncology Group (COG)—that develop new treatments for children with brain tumors, share information, and have common goals. The Pediatric Brain Tumor Consortium (PBTC) is a second NCI-sponsored organization that has clinical trials specifically designed for children with brain tumors. You may be referred to a children’s hospital or academic medical center for participation in a clinical trial sponsored by either group.

Research is important for finding and providing new or improved treatments. Your child may be the first to receive new therapies before they are more widely available; they often become standard treatment. By evaluating new therapies for large numbers of children through COG or PBTC, researchers can more quickly and efficiently gather information about effective therapies.

Phases of Clinical Trials: Clinical trials are often described as being phase I, phase II, or phase III. Phase I trials are done to evaluate the side effects of a new treatment and to establish the proper dose. Different patients may receive different doses of the same medicine. Although doctors hope that the treatment may help the patient, that is not the main goal of a phase I clinical trial. After a phase I trial has been completed and the proper dose of the new medicine has been determined, a phase II trial may begin. In a phase II trial, all of the patients receive the same dose of the medicine and the goal is to see how effective the new treatment will be. If a phase II trial finds that the new treatment is very promising, a phase III trial may be done. In a phase III trial, patients are randomly given one of two different treatments. Randomly means that a computer (not the doctor or parent) decides which of the treatments a given patient will receive. A phase III trial is usually done to find out whether a new treatment is better than, worse than, or the same as the established treatment for a certain disease.


How to Find Clinical Trials: More information on clinical trials can be obtained through:


Pediatric Brain Tumor Constortium offers their available phase 1 and 2 clinical trial information at


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