The Myeloma Alphabet Soup Handbook
Clinical Trials

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Phase 1 Trials
The purpose of a phase I study is to find the best way to give a new treatment and how much of it can be given safely. In a phase I study, a new treatment is given to a small number of patients. For a new drug, the study starts by giving a very low dose of the drug, then the dose is slowly increased as new patients enter the trial. The dose can be increased by giving more at one time or by giving the same dose more often. Physicians watch patients carefully for any harmful side effects. Although the research treatment has been well tested in laboratory and animal studies, the side effects in patients can not be completely known ahead of time. Phase I studies may involve significant risks for this reason. They are offered only to patients whose cancer cannot be helped by other known treatments. Phase I treatments may or may not produce anticancer effects, but some patients have been helped by these treatments. Once the best dose is chosen, the drug is studied for its ability to shrink tumors in phase II trials.

Phase 2 Trials
Phase II studies are designed to find out if the treatment actually kills cancer cells in people. Usually groups of 20 to 50 patients with one type of cancer receive a phase II treatment. For example, patients with breast cancer that no longer responds to accepted therapy (it has become resistant to standard therapy) may be treated on a phase II study. Patients are closely observed for anticancer effect by repeated measurement of tumor size to see if it has shrunk since the beginning of the study. When the tumor gets a lot smaller and stays smaller for at least a month, the patient is said to have "responded" to the treatment. If at least one-fifth of the patients in the phase II study respond to treatment, the treatment is judged active against their tumor type. In addition to monitoring patients for response, any side effects of the treatment are carefully recorded and assessed. Since larger numbers of patients receive the treatment in phase II studies than in phase I studies, there is more chance to observe unusual side effects. Each new phase of a clinical trial depends on and builds on information from an earlier phase. If a treatment has shown activity against cancer in a phase II study, it becomes part of a phase III study.

Phase 3 Trials
Phase III studies usually compare standard treatments (the treatment most accepted) with treatments that appeared to be good in the small phase II studies. Phase III studies require large numbers of patients; some studies use thousands of patients. Patients are usually randomized, which means they are assigned by chance to one of the treatments being studied. The group that receives the standard treatment is called the "control" group. The researchers know that a certain number of these patients will be helped by the treatment. Another patient group receives the newer therapy to see if it will help the patients more. Phase III studies look for longer life, better quality of life, fewer side effects, and fewer cases of the cancer returning.

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Last Updated: 03/27/01