An unintended reaction to a drug taken at doses normally used in man for prophylaxis, diagnosis, or therapy of disease, or for the modification of physiological function. In clinical trials, an ADR would include any injuries by overdosing, abuse/dependence, and unintended interactions with other medicinal products.

A negative experience encountered by an individual during the course of a clinical trial, that is associated with the drug. An AE can include previously undetected symptoms, or the exacerbation of a pre-existing condition. When an AE has been determined to be related to the investigational product, it is considered an Adverse Drug Reaction.

Molecules that bond to the receptor site of a protein. Unlike agonists, antagonists suppress or inhibit the function of the protein to which they bind.

A component of the body's immune response. A Y-shaped protein, it is secreted in response to an antigenic stimulus. It neutralizes the antigen it by binding to it.

See Tumor Necrosis Factor.

The cell's self-destruct mechanism, also known as "programmed cell death." In this process, the cell dies via "self digestion," without rupturing and releasing its contents into the surrounding tissue. Without normal cell apoptosis, cells can grow uncontrollably, causing cancer.

Any of the treatment groups in a randomized trial. Most randomized trials have two "arms"; some have three or more. Open-label studies have one arm.

A pharmacokinetic measure of drug exposure over time.

A pharmacokinetic measure adjusting for baseline drug levels.

Information gathered at the beginning of a study from which variations found in the study are measured; a known value or quantity with which an unknown is compared when measured or assessed; and/or the initial time point in a clinical trial, just before subjects begin receiving the experimental treatment being tested.

A virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, or analogous product applicable to the prevention, treatment or cure of diseases or injuries of man.

Any technique that uses living organisms, or substances from organisms, biological systems, or processes to make or modify a product or process, to change plants or animals, or to develop micro-organisms for specific uses.

The process through which one or more parties to a clinical trial are unaware of the treatment assignments. In a single-blinded study, usually the subjects are unaware of the treatment assignments. In a double-blinded study, both the subjects and the investigators are unaware of the treatment assignments.

A randomized trial is "blind" if subjects and study personnel are not told which arm of the trial the patient is in. Blinded studies are used to prevent bias.

Diseases resulting from unregulated growth among the body's own cells. The growth is caused by mutations in the genetic material of cancerous cells.

A "complementary" DNA used in the identification of expressed genes.

The cycle of DNA replication and cellular division that a cell progresses through in order to divide into two cells. It is thought that understanding more about the cell cycle could lead scientists to a better understanding of cancer.

The tendency of cells, in particular cancer cells, to divide indefinitely.

Any therapy using chemically-synthesized drugs; more commonly, the application of such methods in the treatment of cancer.

A research study to answer specific questions about vaccines, new treatments, or new ways of using known treatments. Clinical trials are used to determine the benefit/risk profile of new medicines or treatments. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people.

The replication of a DNA sequence from one organism to another through genetic engineering.

A word for an individual amino acid. Each codon is a string of exactly three bases. A gene is a sequence comprised entirely of codons.

The CHMP is responsible for preparing the European Medicine Agency's opinions on all questions concerning medicinal products for human use and plays a vital role in the marketing procedures for medicines in the European Union.

A document explaining all relevant study information to assist the study volunteer in understanding the expectations and requirements of participation in a clinical trial. This document is presented to and signed by the study subject.

The standard by which experimental observations are evaluated. In many clinical trials, one group of subjects will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

The contents of the cell's protoplasm apart from the nucleus.

One of the four bases found in DNA. It pairs with guanine.

The process of handling the data gathered during a clinical trial. May also refer to the department responsible for managing data entry and database generation and/or maintenance.

A series of guidelines adopted by the 18th World Medical Assembly in Helsinki, Finland in 1964. The Declaration addresses ethical issues for physicians conducting biomedical research involving human subjects.

Refers to the characteristics of study participants, including sex, age, family medical history, and other characteristics relevant to the study in which they are enrolled.

A process induced by heat or chemicals in which a biomolecule (e.g. DNA, RNA or a protein) loses its natural form.

A clinical trial in which two or more doses of an agent are tested against each other to determine which dose works best and is least harmful.

A clinical trial design in which neither the participating subjects nor the study staff knows which subjects are receiving the experimental medicine and which are receiving a placebo or another therapy.

As defined by the Food, Drug and Cosmetic Act, drugs are "articles (other than food) intended for the use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals, or to affect the structure or any function of the body of man or other animals."

A finished dosage form (e.g. tablet, capsule, or solution) that contains the active drug ingredient usually combined with inactive ingredients.

A modification of the effect of a medicine when administered with another medicine. The effect may be an increase or decrease in the action of either substance, or it may be an adverse event not normally associated with either medicine.

The dose of an investigational agent that produces the outcome considered "effective," as defined in the study protocol.

The ability of a medicine or treatment to produce a result. A medicine passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed.

A decentralized body of the European Union responsible for the scientific evaluation of applications for marketing authorization for medicinal products.

Overall outcome that the protocol is designed to evaluate. Common endpoints are efficacy endpoints, treatment outcomes, severe toxicity, disease progression, or death.

The branch of medical science that deals with the study of incidence and distribution and control of a disease in a population or sub-population.

An independent group of both medical and non-medical professionals who are responsible for verifying the integrity of a study and ensuring the safety, integrity, and human rights of the study participants.

A decentralized body of the European Union with headquarters in London. Its main responsibility is the protection and promotion of public and animal health, through the evaluation and supervision of medicines for human and veterinary use.

Department within the United States Department of Health and Human Services. Enforces Food, Drug and Cosmetics Act and related federal public health laws. Grants IND, IDE, PMA and NDA approvals.

The mixture of chemicals and/or biological substances and excipients used to prepare dosage forms.

Functional segments of our genetic material that serve mostly as blueprints for the synthesis of proteins.

The study of inheritance; deals with the laws of inheritance and the properties of genes, including the transmission of specific variants of a gene from one generation to the next.

The study of genes, and how they affect the structure, growth, health, and diseases of an organism.

A benign or malignant brain and spinal cord tumor that arises from glial cells (astrocytes, oligodendrocytes, ependymal cells).

The process by which ogliosaccharide units are added to proteins in order to form different chemical bonds.

International ethical and scientific quality standard for designing, conducting, monitoring, recording, auditing, analyzing and reporting studies.

A protein that stimulates the production of scavenging cells in the immune system.

One of the four bases found in DNA. It pairs with cytosine.

A patient or healthy individual participating in a research study. A living individual about whom an investigator obtains private information or data through intervention or interaction.

The medical or social standards determining whether a subject may be chosen to enter a clinical trial or epidemiologic study.

A valid reason to use a certain test, medication, procedure, or surgery.

An independent body constituted of medical/scientific professionals and non-medical/nonscientific members, whose responsibility it is to ensure the protection of the rights, safety and well-being of human subjects involved in a trial.

A process by which a subject voluntarily confirms his or her willingness to participate in a particular trial, after having been informed of all aspects of the trial that are relevant to the subject's decision to participate.

Location of research. Retains ultimate responsibility for human subject regulation compliance.

An independent group of professionals designated to review and approve the clinical protocol, informed consent forms, study advertisements, and patient brochures, to ensure that the study is safe and effective for human participation.

The petition through which a drug sponsor requests the FDA to allow human testing of its drug product.

A medical professional, usually a physician but may also be a nurse, pharmacist or other health care professional, under whose direction an investigational drug is administered or dispensed.

Relevant clinical and non-clinical data compiled on the investigational drug, biologic or device being studied.

Non-clinical testing conducted in an artificial environment such as a test tube or culture medium.

Testing conducted in living animal and human systems.

A molecule or ion having the ability to bond with a protein.

The transformation of chemical substances in the body or within a cell.

A widely-used synonym for DNA chip.

Messenger RNA, the working copy of a gene that acts as a blueprint for the synthesis of proteins. Unlike DNA, it is able to leave the cell nucleus.

Generic chemical term for DNA and RNA; chain-shaped molecules whose individual building blocks are bases (nucleotides).

The building blocks of DNA and RNA; they comprise the four bases adenine, thymine, cytosine and guanine (A, T, C, G; in RNA thymine is replaced by uracil [U]), a sugar and at least one phosphate group.

The unauthorized use of a drug for a purpose other than that approved of by a regulatory body such as the FDA or EMA.

Short nucleic acid chains composed of at most a few dozen building blocks (nucleotides).

A gene that plays a role in the development of cancer.

The medical science relating to the development, diagnosis, treatment, and prevention of tumors.

A study in which doctors and participants know which medicine or vaccine is being administered.

A designation of the FDA to indicate a therapy developed to treat a rare disease (one which afflicts a U.S. population of less than 200,000 people).

Studies of the end results of particular health care practices and interventions, including effects that subjects experience and care about.

Drugs available for purchase without a physician's prescription.

The study of cost-benefit ratios of drugs with other therapies or with similar drugs. Also referred to as "outcomes research."

Describes the influence of gene variations in individuals on the efficacy and side effects of drugs.

Studies interactions between drugs and the genome.

The processes in a living organism of absorption, distribution, metabolism, and excretion of a medicine or vaccine.

Phase I studies are designed to allow scientists and medical doctors to understand what effects an investigational compound has in human subjects. The goal is to study what happens to the investigational compound in the body from the time it is swallowed or injected until it is excreted.

Phase II studies are designed to evaluate the safety and efficacy of an investigational compound in patients with a specific disease or condition. Patients are given various doses of the compound and closely monitored to compare the effects and to determine the safest dosing regimen.

Phase III studies are designed to confirm the safety and efficacy of an investigational compound, and the dosage regimen chosen, in large numbers of patients with a specific disease or condition.

Phase IV studies take place after the drug has been approved for marketing and are designed to provide broader experience in evaluating the safety and effectiveness of the new medicine in larger numbers of patients.

Usually a phase III study that presents the data a governing body (e.g. the FDA) uses to decide whether or not to approve a drug.

An inactive substance designed to resemble the drug being tested. It is used as a control to rule out any psychological effects testing may present.

A method of medicine investigation in which an inactive substance (the "placebo") is given to one group of subjects, while the test medicine is given to another group.

A technique for rapid copying (amplification) of even minute amounts of DNA.

Enzymes that link individual nucleotides together to form long DNA or RNA chains.

Existence in more than one form; in genetics, a region of DNA in which differences in the sequence of building blocks occur in a relatively large number of people.

Before a drug may be tested on humans, pre-clinical studies must be conducted either in vitro but usually in vivo on animals to determine that the drug is safe.

Ancestor cells that can restore function in tissues damaged by disease.

A molecule consisting of a chain of amino acids. Because of the variety of their building blocks, proteins can differ greatly in form and function.

The study of the form, function and interactions of all the proteins of a tissue or organism.

A detailed plan that sets forth the objectives, study design, and methodology for a clinical trial. A study protocol must be approved by an IRB before investigational drugs may be administered to humans.

Systems and procedures designed to ensure that a study is being performed in compliance with Good Clinical Practice (GCP) guidelines and that the data being generated is accurate.

Exploration of ways to measure comfort and quality of life for individuals with chronic illnesses.

A method based on chance by which study subjects are assigned to a treatment group. The method minimizes differences among groups by equally distributing people with particular characteristics among all trial arms.

In clinical trials, the department or function that is responsible for ensuring compliance with government regulations and interacts with the regulatory agencies.

Risk to individual subject vs. potential benefits. Also called Risk-Benefit Analysis.

Any adverse event (AE) that is fatal, life-threatening, permanently disabling, or which results in hospitalization, initial or prolonged.

A pathological condition occurring by consequence of a disease.

A measurement of the persistence of serum, the fluid created when blood is separated into its solid and liquid components. Half-life is the amount of time that elapses before half a sample is metabolized or eliminated by normal biological processes.

Any undesired action or effect of a medicine or treatment. Experimental medicine must be evaluated for both immediate and long-term effects.

The movement of signals from the outside of the cell to the inside. Scientists are attempting to learn more about this process in cancer cells in order to fight the disease.

A study in which one party, either the investigator or subject, does not know the medication being taken by the subject.

Combinations of small molecules. Creating new small molecule compounds is often a step in drug discovery.

The building blocks of nucleic acids, such as amino acids, vitamins, purines, and pyrimidines.

Differences in individual building blocks (base pairs) of DNA that are distributed randomly over the genome and passed from generation to generation.

Individual, company, institution or organization taking responsibility for initiation, management and financing of study.

Official, detailed, written instructions for the management of clinical trials. SOPs ensure that all the functions and activities of a clinical trial are carried out in a consistent and efficient manner.

The currently accepted treatment or intervention considered to be effective in the treatment of a specific disease or condition.

The probability that an event or difference occurred by chance alone. In clinical trials, the level of statistical significance depends on the number of subjects studied and observations made.

A primary or secondary outcome used to judge the effectiveness or safety of a treatment.

A study designed to obtain additional safety and/or efficacy data. These studies may be clinical trials or non-interventional, and are often conducted as a post marketing (Phase 4) activity.

The molecules, mostly proteins, upon which drugs act in our body.

The medical treatment of disease.

One of the four bases found in DNA. It pairs with adenine.

An adverse event produced by a medicine that is detrimental to the subject's health. The level of toxicity associated with a medicine varies with the condition for which the medicine is used.

The process by which enzymes use the genetic information on a strand of DNA to create a complimentary strand of messenger RNA (mRNA).

Molecules that carry amino acids during the process of protein synthesis.

The process of transferring DNA from a donor to a recipient cell.

A term describing an organism containing genetic material from a source other than its parents.

Animals containing genes derived from other species.

A protein originally shown to have the potential to kill cancerous tumors, and an important cytokine in the pathogenesis of RA.

A molecule which, when functioning correctly, prevents cancer from developing.

A reaction that is not consistent in nature or severity with study application.