Glossary of Terms
Adverse Reaction: (Adverse Event.) An unwanted effect caused by the administration of drugs.
Onset may be sudden or develop over time (See Side Effects).
Advocacy and Support Groups: Organizations and groups that actively support participants and their
families with valuable resources, including self-empowerment and survival tools.
Approved Drugs: In the U.S., the Food and Drug Administration (FDA) must approve a substance
as a drug before it can be marketed. The approval process involves several
steps including pre-clinical laboratory and animal studies, clinical trials
for safety and efficacy, filing of a New Drug Application by the manufacturer
of the drug, FDA review of the application and FDA approval/rejection
of application (See Food and Drug Administration).
ARM: Any of the treatment groups in a randomized trial. Most randomized trials
have two "arms," but some have three "arms" or even
more (See Randomized Trial).
Baseline: 1. Information gathered at the beginning of a study from which variations
found in the study are measured. 2. A known value or quantity with which
an unknown is compared when measured or assessed. 3. The initial time
point in a clinical trial, just before a participant starts to receive
the experimental treatment which is being tested. At this reference point,
measurable values such as CD4 count are recorded. Safety and efficacy
of a drug are often determined by monitoring changes from the baseline values.
Bias: When a point of view prevents impartial judgment on issues relating to
the subject of that point of view. In clinical studies, bias is controlled
by blinding and randomization (See Blind and Randomization).
Blind: A randomized trial is "Blind" if the participant is not told
which arm of the trial he is on. A clinical trial is "Blind"
if participants are unaware of whether they are in the experimental or
control arm of the study. This is also called masked. (See Single Blind
Study and Double Blind Study).
Clinical: Pertaining to or founded on observation and treatment of participants,
as distinguished from theoretical or basic science.
Clinical Endpoint: See Endpoint.
Clinical Investigator: A medical researcher in charge of carrying out a clinical trial's protocol.
Clinical Trial: A clinical trial is a research study to answer specific questions about
vaccines or new therapies or new ways of using known treatments. Clinical
trials (also called medical research and research studies) are used to
determine whether new drugs or treatments are both safe and effective.
Carefully conducted clinical trials are the fastest and safest way to
find treatments that work in people. Trials are in four phases: Phase
I tests a new drug or treatment in a small group; Phase II expands the
study to a larger group of people; Phase III expands the study to an even
larger group of people; and Phase IV takes place after the drug or treatment
has been licensed and marketed. (See Phase I, II, III, and IV Trials).
Cohort: In epidemiology, a group of individuals with some characteristics in common.
Community-based Clinical Trial (CBCT): A clinical trial conducted primarily through primary-care physicians rather
than academic research facilities.
Compassionate Use: A method of providing experimental therapeutics prior to final FDA approval
for use in humans. This procedure is used with very sick individuals who
have no other treatment options. Often, case-by-case approval must be
obtained from the FDA for "compassionate use" of a drug or therapy.
Complementary and Alternative Therapy: Broad range of healing philosophies, approaches, and therapies that Western
(conventional) medicine does not commonly use to promote well-being or
treat health conditions. Examples include acupuncture, herbs, etc. Internet Address:
Confidentiality Regarding Trial Participants: Refers to maintaining the confidentiality of trial participants including
their personal identity and all personal medical information. The trial
participants' consent to the use of records for data verification
purposes should be obtained prior to the trial and assurance must be given
that confidentiality will be maintained.
Contraindication: A specific circumstance when the use of certain treatments
could be harmful.
Control: A control in randomized controlled trials refers to studying a group
of treated patients not in isolation but in comparison to other groups
of patients, the control groups, who by not receiving the treatment under
study give investigators important clues to the effectiveness of the treatment,
its side effects, and the parameters that modify these effects.
Control Group: The standard by which experimental observations are evaluated. In many
clinical trials, one group of patients will be given an experimental drug
or treatment, while the control group is given either a standard treatment
for the illness or a placebo (See Placebo and Standard Treatment).
Controlled Trials: Control is a standard against which experimental observations may be
evaluated. In clinical trials, one group of participants is given an experimental
drug, while another group (i.e., the control group) is given either a
standard treatment for the disease or a placebo.
Data Safety and Monitoring Board (DSMB): An independent committee composed of community representatives and clinical
research experts, that reviews data while a clinical trial is in progress
to ensure that participants are not exposed to undue risk. A DSMB may
recommend that a trial be stopped if there are safety concerns or if the
trial objectives have been achieved.
Diagnostic Trials: Refers to trials that are conducted to find better tests or procedures
for diagnosing a particular disease or condition. Diagnostic trials usually
include people who have signs or symptoms of the disease or condition
Dose-Ranging Study: A clinical trial in which two or more doses of an agent (such as a drug)
are tested against each other to determine which dose works best and is
Double-Blind Study: A clinical trial design in which neither the participating individuals
nor the study staff knows which participants are receiving the experimental
drug and which are receiving a placebo (or another therapy). Double-blind
trials are thought to produce objective results, since the expectations
of the doctor and the participant about the experimental drug do not affect
the outcome; also called double-masked study. See Blinded Study, Single-Blind
Study, and Placebo.
Double-Masked Study: See Double-Blind Study.
Drug-Drug Interaction: A modification of the effect of a drug when administered with another
drug. The effect may be an increase or a decrease in the action of either
substance, or it may be an adverse effect that is not normally associated
with either drug.
DSMB: See Data Safety and Monitoring Board.
Efficacy: (Of a drug or treatment). The maximum ability of a drug or treatment
to produce a result regardless of dosage. A drug passes efficacy trials
if it is effective at the dose tested and against the illness for which
it is prescribed. In the procedure mandated by the FDA, Phase II clinical
trials gauge efficacy and Phase III trials confirm it (See Food and Drug
Administration (FDA), Phase II and III Trials).
Eligibility Criteria: Summary criteria for participant selection; includes Inclusion and Exclusion
criteria. (See Inclusion/Exclusion Criteria)
Empirical: Based on experimental data, not on a theory.
Endpoint: Overall outcome that the protocol is designed to evaluate. Common endpoints
are severe toxicity, disease progression or death.
Enrolling: The act of signing up participants into a study. Generally this process
involves evaluating a participant with respect to the eligibility criteria
of the study and going through the informed consent process.
Epidemiology: The branch of medical science that deals with the study of incidence and
distribution and control of a disease in a population.
Exclusion/Inclusion Criteria: See Inclusion/Exclusion Criteria.
Expanded Access: Refers to any of the FDA procedures, such as compassionate use, parallel
track and treatment IND that distribute experimental drugs to participants
who are failing on currently available treatments for their condition
and also are unable to participate in ongoing clinical trials.
Experimental Drug: A drug that is not FDA licensed for use in humans, or as a treatment for
a particular condition (See Off-Label Use).
FDA: See Food and Drug Administration.
Food and Drug Administration (FDA): The U.S. Department of Health and Human Services agency responsible for
ensuring the safety and effectiveness of all drugs, biologics, vaccines
and medical devices, including those used in the diagnosis, treatment
and prevention of HIV infection, AIDS and AIDS-related opportunistic infections.
The FDA also works with the blood banking industry to safeguard the nation's
blood supply. Internet address:
Hypothesis: A supposition or assumption advanced as a basis for reasoning or argument,
or as a guide to experimental investigation.
Inclusion/Exclusion Criteria: The medical or social standards determining whether a person may or may
not be allowed to enter a clinical trial. These criteria are based on
such factors as age, gender, the type and stage of a disease, previous
treatment history, and other medical conditions. It is important to note
that inclusion and exclusion criteria are not used to reject people personally,
but rather to identify appropriate participants and keep them safe.
IND: See Investigational New Drug.
Informed Consent: The process of learning the key facts about a clinical trial before deciding
whether or not to participate. It is also a continuing process throughout
the study to provide information for participants. To help someone decide
whether or not to participate, the doctors and nurses involved in the
trial explain the details of the study.
Informed Consent Document: A document that describes the rights of the study participants, and includes
details about the study, such as its purpose, duration, required procedures
and key contacts. Risks and potential benefits are explained in the informed
consent document. The participant then decides whether or not to sign
the document. Informed consent is not a contract, and the participant
may withdraw from the trial at any time.
Institutional Review Board (IRB): 1) A committee of physicians, statisticians, researchers, community advocates,
and others that ensures that a clinical trial is ethical and that the
rights of study participants are protected. All clinical trials in the
U.S. must be approved by an IRB before they begin. 2) Every institution
that conducts or supports biomedical or behavioral research involving
human participants must, by federal regulation, have an IRB that initially
approves and periodically reviews the research in order to protect the
rights of human participants.
Intent to Treat: Analysis of clinical trial results that includes all data from participants
in the groups to which they were randomized (See Randomization) even if
they never received the treatment.
Intervention Name: The generic name of the precise intervention being studied.
Interventions: Primary interventions being studied; types of interventions are Drug, Gene
Transfer, Vaccine, Behavior, Device, or Procedure.
Investigational New Drug: A new drug, antibiotic drug, or biological drug that is used in a clinical
investigation. It also includes a biological product used in vitro for
IRB: See Institutional Review Board.
Masked: The knowledge of intervention assignment. See Blind
Natural History Study: Study of the natural development of something (such as an organism or
a disease) over a period of time.
New Drug Application (NDA): An application submitted by the manufacturer of a drug to the FDA - after
clinical trials have been completed - for a license to market the drug
for a specified indication.
Off-Label Use: A drug prescribed for conditions other than those approved by the FDA.
Open-Label Trial: A clinical trial in which doctors and participants know which drug or
vaccine is being administered.
Orphan Drugs: An FDA category that refers to medications used to treat diseases and
conditions that occur rarely. There is little financial incentive for
the pharmaceutical industry to develop medications for these diseases
or conditions. Orphan drug status, however, gives a manufacturer specific
financial incentives to develop and provide such medications.
Peer Review: Review of a clinical trial by experts chosen by the study sponsor. These
experts review the trials for scientific merit, participant safety, and
Pharmacokinetics: The processes (in a living organism) of absorption, distribution, metabolism
and excretion of a drug or vaccine.
Phase I Trials: Initial studies to determine the metabolism and pharmacologic actions
of drugs in humans, the side effects associated with increasing doses,
and to gain early evidence of effectiveness; may include healthy participants
Phase II Trials: Controlled clinical studies conducted to evaluate the effectiveness of
the drug for a particular indication or indications in patients with the
disease or condition under study and to determine the common short-term
side effects and risks.
Phase III Trials: Expanded controlled and uncontrolled trials after preliminary evidence
suggesting effectiveness of the drug has been obtained, and are intended
to gather additional information to evaluate the overall benefit-risk
relationship of the drug and provide an adequate basis for physician labeling.
Phase IV Trials: Post-marketing studies to delineate additional information including the
drug's risks, benefits and optimal use.
Placebo: A placebo is an inactive pill, liquid or powder that has no treatment
value. In clinical trials, experimental treatments are often compared
with placebos to assess the treatment's effectiveness. (See Placebo
Placebo Controlled Study: A method of investigation of drugs in which an inactive substance (the
placebo) is given to one group of participants, while the drug being tested
is given to another group. The results obtained in the two groups are
then compared to see if the investigational treatment is more effective
in treating the condition.
Placebo Effect: A physical or emotional change, occurring after a substance is taken or
administered, that is not the result of any special property of the substance.
The change may be beneficial, reflecting the expectations of the participant
and, often, the expectations of the person giving the substance.
Preclinical: Refers to the testing of experimental drugs in the test tube or in animals
- the testing that occurs before trials in humans may be carried out.
Prevention Trials: Refers to trials that find better ways to prevent disease in people who
have never had the disease or to prevent a disease from returning. These
approaches may include medicines, vaccines, vitamins, minerals, or lifestyle changes.
Protocol: A study plan on which all clinical trials are based. The plan is carefully
designed to safeguard the health of the participants as well as answer
specific research questions. A protocol describes what types of people
may participate in the 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.
Quality of Life Trials (or supportive care trials): Refers to trials that explore ways to improve
comfort and quality of life for individuals with a chronic illness.
Randomization: A method based on chance by which study participants are assigned to
a treatment group. Randomization minimizes the differences among groups
by equally distributing people with particular characteristics among all
the trial arms. The researchers do not know which treatment is better.
From what is known at the time, any one of the treatments chosen could
be of benefit to the participant (See Arm).
Randomized Trial: A study in which participants are randomly (i.e., by chance) assigned
to one of two or more treatment arms of a clinical trial. Occasionally
placebos are utilized. (See Arm and Placebo).
Recruiting: The period during which a trial is attempting to identify and enroll
participants. Recruitment activities can include advertising and other
ways of soliciting interest from possible participants. (See recruitment
status and enrolling).
Recruitment Status: Indicates the current stage of a trial, whether it is planned, ongoing
or completed. Possible values include:
- Not yet recruiting: participants are not yet being recruited or enrolled
- Recruiting: participants are currently being recruited and enrolled
- Enrolling by invitation: participants are being (or will be) selected from
a predetermined population
- Active, not recruiting: study is ongoing (i.e., patients are being treated
or examined), but enrollment has completed
- Completed: the study has concluded normally; participants are no longer
being examined or treated (i.e., last patient's last visit has occurred)
- Suspended: recruiting or enrolling participants has halted prematurely
but potentially will resume
- Terminated: recruiting or enrolling participants has halted prematurely
and will not resume; participants are no longer being examined or treated
- Withdrawn: study halted prematurely, prior to enrollment of first participant
Risk-Benefit Ratio: The risk to individual participants versus the potential benefits. The
risk/benefit ratio may differ depending on the condition being treated.
Screening Trials: Refers to trials which test the best way to detect certain diseases or
Side Effects: Any undesired actions or effects of a drug or treatment. Negative or adverse
effects may include headache, nausea, hair loss, skin irritation or other
physical problems. Experimental drugs must be evaluated for both immediate
and long-term side effects (See Adverse Reaction).
Single-Blind Study: A study in which one party, either the investigator or participant, is
unaware of what medication the participant is taking; also called single-masked
study. (See Blind and Double-Blind Study).
Single-Masked Study: See Single-Blind Study.
Standard Treatment: A treatment currently in wide use and approved by the FDA, considered
to be effective in the treatment of a specific disease or condition.
Standards of Care: Treatment regimen or medical management based on state of the art participant care.
Statistical Significance: The probability that an event or difference occurred by chance alone.
In clinical trials, the level of statistical significance depends on the
number of participants studied and the observations made, as well as the
magnitude of differences observed.
Study Endpoint: A primary or secondary outcome used to judge the effectiveness of a treatment.
Study Type: The primary investigative techniques used in an observational protocol;
types are Purpose, Duration, Selection and Timing.
Suspended: See Recruitment Status
Terminated: See Recruitment Status
Toxicity: An adverse effect produced by a drug that is detrimental to the participant's
health. The level of toxicity associated with a drug will vary depending
on the condition which the drug is used to treat.
Treatment IND: IND stands for Investigational New Drug application, which is part of
the process to get approval from the FDA for marketing a new prescription
drug in the U.S. It makes promising new drugs available to desperately
ill participants as early in the drug development process as possible.
Treatment INDs are made available to participants before general marketing
begins, typically during Phase III studies. To be considered for a treatment
IND a participant cannot be eligible to be in the definitive clinical trial.
Treatment Trials: Refers to trials which test new treatments, new combinations of drugs
or new approaches to surgery or radiation therapy.
Withdrawn: See Recruitment Status
Source: ClinicalTrials.gov (A Service of the U.S. National Institutes of Health)