FAQ Clinical Trials

  • What is a Clinical trial?  A trial where patients receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants' behavior.  Clinical trials may compare a new medical approach to a standard one that is already available or to a placebo that contains no active ingredients or to no intervention. Some clinical trials compare interventions that are already available to each other. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants.

  • What is a Protocol?  A study plan on which the clinical trial is based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes who may participate in a specific research trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

  • What is a Phase I trial? A trial that helps determine dose scheduling, toxicity, safety, and feasibility of a treatment. Its purpose is to determine whether a drug or treatment will be safe for humans and whether it appears to function as expected. Most phase I trials are not designed to answer whether a treatment is effective. Side effects are closely monitored.

  • What is a Phase II trial?  A trial performed to further evaluate safety and assess the therapeutic effectiveness of the new treatment in specific forms of cancer. These trials are typically larger than Phase I trials and usually include 100-200 patients. Phase II trial results provide insight into which cancers may be most effectively treated with a new therapy.

  • What is a Phase III trial? A trial that is carried out once efficacy is established.  This type of trial compares the new drug treatment to an existing standard drug treatment for the same diagnosis.  These trials are often quite large with many thousands of patients participating.  These studies are often randomized, double-blind, and placebo-controlled.  The results of these trials may change the standard of care if the new treatment is effective.

  • What is a Phase IV trial? These large trials are usually done after the drug has FDA approval. They are designed so the drug is used as standard therapy to determine optimal use of the drug for large patient populations

  • What is a placebo? An inactive intervention designed to resemble, as much as possible, its counterpart in clinical research.

  • What does placebo-controlled mean?  These are trials that compare a new treatment to a placebo, or inactive treatment or substance.

  • What does randomized mean? This means that patients are randomly assigned to one treatment or another.

  • What does double-blinded mean? This means that the study patients and investigators are not aware of which treatment is being given to any one individual until after the study is completed.