Glossary of Terms

This information is provided by the National Cancer Institute. Cancer Clinical Trials: The Basic Workbook

Adverse effect: See side effects.

Agent: In a cancer clinical trial, an agent is a substance that produces, or is capable of producing, an effect that fights cancer.

Anthracycline: A member of a family of anticancer drugs that are also antibiotics.

Bias: Human choices, beliefs, or any other factors besides those being studied that affect a clinical trial’s results. Clinical trials use many methods to avoid bias because biased results may not be accurate.

Biological therapy: Treatment to stimulate or restore the ability of the immune system to fight infection and disease. Also used to lessen side effects that may be caused by some cancer treatments.
Also known as immunotherapy, biotherapy, or biological response modifier (BRM) therapy.

Cancer: A term for diseases in which abnormal cells divide without control. Cancer cells can invade nearby tissues and can spread through the bloodstream and lymphatic system to other parts of the body.

Cancer vaccine: A form of biological therapy which may help a person’s immune system to recognize cancer cells. These vaccines may help the body reject tumors and prevent cancer from recurring.

Chemoprevention: The use of drugs, vitamins, or other agents to try to reduce the risk of or delay the development or recurrence of cancer.

Chemotherapy: Treatment with anticancer drugs.

Clinical trial: A research study that tests how well new medical treatments or other interventions work in people. Each study is designed to test new methods of screening, prevention, diagnosis, or treatment of a disease.

Community Clinical Oncology Program (CCOP): A program that links community physicians with NCI clinical research programs, enabling more people with cancer to participate in clinical trials in their own communities.

Control group: In a randomized clinical trial, the group that receives standard treatment.

Cooperative groups: Networks of institutions that jointly carry out large clinical trials following the same protocols.

Data and safety monitoring board (DSMB): An independent committee whose membership includes, at minimum, a statistician and a clinical expert in the area being studied. Responsibilities of the DSMB are to: ensure that risks associated with participation are minimized to the extent possible, ensure the integrity of the
data, and stop a trial either if safety concerns arise or as soon as its objectives have been met.

Diagnostic trial: A research study that evaluates methods of detecting disease.

Eligibility criteria: Participant eligibility criteria for clinical trials can range from general (age, sex, type of cancer) to specific (prior treatment, tumor characteristics, blood cell counts, organ function). Eligibility criteria may also vary with trial phase. In phase 1 and 2 trials, the criteria often focus on making sure that people who might be harmed because of abnormal organ function or other factors are not put at risk. Phase 2 and 3 trials often add criteria regarding disease type and stage, and number of prior treatments.

Endpoint: What researchers measure to evaluate the results of a new treatment being tested in a clinical trial. Research teams establish the endpoints of a trial before it begins. Examples of endpoints include toxicity, tumor response, survival time, and quality of life.

Food and Drug Administration (FDA): An agency of the U.S. Department of Health and Human Services whose mission it is to promote and protect the public health:
  • by ensuring that medical products are proven safe and effective before they can be used by patients and
  • by monitoring products for continued safety after they are in use.

Gene: The functional and physical unit of heredity passed from parent to offspring. Genes are pieces of DNA, and most genes contain the information for making a specific protein.

Gene therapy: Treatment that alters a gene. In studies of gene therapy for cancer, researchers are trying to improve the body’s natural ability to fight the disease or to make the cancer cells more sensitive to other kinds of therapy.

Genetic: Inherited; having to do with information that is passed from parents to offspring through genes in sperm and egg cells.

Genetic testing: Analyzing DNA to look for a genetic alteration that may indicate an increased risk for developing a specific disease or disorder.

Genetics research: Research that focuses on how someone’s genetic makeup can assist in the early detection, diagnosis, or treatment of cancer. Genetics research may be a part of screening or treatment trials.

Investigation group: In a clinical trial, the group that receives the new agent being tested. See also control group.

Imaging: Tests that produce pictures of areas inside the body.

Immune system: The complex group of organs and cells that defends the body against infection or disease.

Immunotherapy: See biological therapy.

Informed consent: The process of providing all relevant information about the trial’s purpose, risks, benefits, alternatives, and procedures to a potential participant, who then, consistent with his or her own interests and circumstances, makes an informed decision about whether to participate.

Institutional review board (IRB): A board designed to oversee the research process in order to protect participant safety. Made up of researchers, ethicists, and lay people from the community, the board must review the trial protocols and the informed consent forms participants sign.

Investigational new drug (IND): A drug that the Food and Drug Administration (FDA) allows to be used in clinical trials, but that the FDA has not yet approved for commercial marketing.

Monoclonal antibodies: A form of biological therapy that may help the body’s own immune system fight cancer by locating cancer cells and either killing them or delivering cancer-killing substances to them without harming normal cells.

Oncology: The branch of medicine that deals with tumors, including study of their development, diagnosis, treatment, and prevention.

National Cancer Institute (NCI): Part of the National Institutes of Health of the United States Department of Health and Human Services, is the Federal Government’s principal agency for cancer research. NCI conducts, coordinates, and funds cancer research, training, health information dissemination, and other programs
with respect to the cause, diagnosis, prevention, and treatment of cancer. Access the NCI Web site at .

New Drug Application (NDA): The application filed with FDA by the trial sponsor once a trial has generated adequate data to support a certain indication for a drug (usually by finding that the drug is safe and superior to standard treatment in a definitive phase 3 trial).

Office for Human Research Protections (OHRP): This office safeguards participants in federally funded research and provides unity and leadership for many federal departments and agencies that carry out research involving human participants.

Phase 1 trial: Small groups of people with cancer are treated with a certain dose of a new agent that has already been extensively studied in the laboratory. During the trial, the dose is usually increased group by group in order to find the highest dose that does not cause harmful side effects. This process determines a safe and appropriate dose to use in a phase 2 trial.

Phase 2 trial: Phase 2 trials continue to test the safety of the new agent and begin to evaluate how well it works against a specific type of cancer. In these trials, the new agent is given to groups of people with one type of cancer or related cancers, using the dosage found to be safe in phase 1 trials.

Phase 3 trial: Phase 3 studies are designed to answer research questions across the disease continuum. Phase 3 trials usually have hundreds to thousands of participants, in order to find out if there are true differences in the effectiveness of the treatment being tested.

Phase 4 trial: Phase 4 trials are used to evaluate the long-term safety and effectiveness of a treatment. Less common than phase 1, 2, and 3 trials, phase 4 trials take place after the new treatment has been approved for standard use.

Physician Data Query (PDQ): PDQ is an online database developed and maintained by the National Cancer Institute. Designed to make the most current, credible, and accurate cancer information available to health professionals and the public, PDQ contains peer-reviewed summaries on cancer treatment, screening, prevention,
genetics, and supportive care; a registry of cancer clinical trials from around the world; and directories of physicians, professionals who provide genetics services, and organizations that provide cancer care.

Placebo: A treatment, often a drug, designed to look like the medicine being tested but that doesn’t contain any active ingredient. Some people call a placebo a “sugar pill.” Placebos are almost never used in cancer treatment trials.

Pre-clinical testing: A process in which scientists test promising new anticancer agents in the laboratory and in animal models. This is done to find out whether agents have an anticancer effect and are safely tolerated in animals. Once a drug proves promising in the lab, the sponsor applies for FDA approval to test it in clinical trials
involving people.

Prevention trials: Trials involving healthy people who are at high risk for developing cancer. These trials try to answer specific questions about and evaluate the effectiveness of ways to reduce the risk of cancer.

Principal investigator (PI): The person, usually a doctor, who is in charge of a clinical trial. The PI prepares a protocol for the trial.

Protocol: A written plan that acts as a “recipe” for conducting a clinical trial. The protocol explains what a trial will do, how it will be carried out, and why each part of the trial is necessary.

Quality of life: The overall enjoyment of life. Many clinical trials measure aspects of an individual’s sense of well-being and ability to perform various tasks to assess the effects of cancer and its treatment on the quality of life.

Radiation therapy: The use of high-energy radiation from x-rays, gamma rays, neutrons, and other sources to kill cancer cells and shrink tumors. Radiation may come from a machine outside the body (external-beam radiation therapy) or it may come from radioactive material placed in the body in the area near cancer cells (internal radiation therapy, implant radiation, or brachytherapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that circulates throughout the body. Also called radiotherapy.

Randomization: A method used to prevent bias in research. A computer generates treatment assignments, and participants have an equal chance to be assigned to one of two or more groups (e.g., the control group or the investigational group).

Randomized clinical trial: A study in which the participants are assigned by chance to separate groups that compare different treatments; neither the researchers nor the participants can choose which group. Using chance to assign people to groups means that the groups will be similar and that the treatments they receive can be compared objectively. At the time of the trial, it is not known which treatment is best. It is the patient’s choice to be in a randomized trial.

Remission: A decrease in or disappearance of signs and symptoms of cancer. In partial remission, some, but not all, signs and symptoms of cancer have disappeared. In complete remission, all signs and symptoms of cancer have disappeared, although there still may be cancer in the body.

Screening: Checking for disease when there are no symptoms.

Screening trials: Clinical trials that focus on what tests can help find cancer in people before they have any cancer symptoms. The goal of early detection/screening trials is to discover new methods for finding cancer as early as possible. For many types of cancer, the ability to find and treat the disease at an early stage provides a
better chance for survival.

Side effects: Problems that occur when treatment affects healthy cells. Common side effects of cancer treatment are fatigue, nausea, vomiting, decreased blood cell counts, hair loss, and mouth sores.

Stage: The extent of a cancer, especially whether the disease has spread from the original site to other parts of the body.

Standard treatment: A currently accepted and widely used treatment for a certain type of cancer, based on the results of past research.

Toxicity: Harmful side effects from an agent being tested.

Treatment trials: Clinical trials conducted to examine new treatment approaches for people who have cancer and to determine the most effective treatments.

Tumor: An abnormal mass of tissue that results from excessive cell division. Tumors perform no useful body function. They may be benign (not cancerous) or malignant (cancerous).

Vaccine: A substance or group of substances meant to cause the immune system to respond to a tumor or to microorganisms, such as bacteria or viruses.

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