Abraham Research Participants
Glossary of Some Clinical Trial Terms
ADVERSE REACTION: (Adverse Event.) An unwanted effect
caused by the administration of drugs. Onset may be sudden or develop over
time.
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.
ARM: Any of the treatment groups in a randomized trial.
Most randomized trials have two "arms," but some have three "arms," or
even more.
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.
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 on whether they are in
the experimental or control arm of the study; also called masked.
CLINICAL: Pertaining to or founded on observation and
treatment of participants, as distinguished from theoretical or basic science.
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.
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.
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 is the nature of the intervention control.
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.
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 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 being studied.
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 least harmful.
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.
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.
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.
ELIGIBILITY CRITERIA: Summary criteria for participant selection;
includes Inclusion and 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.
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.
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: http://www.fda.gov/.
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.
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 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 diagnostic purposes.
MASKED: The knowledge of intervention assignment.
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 ethical considerations.
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 and/or patients.
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 and 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.
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 to 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.
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.
RECRUITING: The period during which a trial is attempting to
identify and enroll participants. Recruitment activites can include advertising
and other ways of solicting interest from possible particpants.
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 health conditions.
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.
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.
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.
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.
SOURCE: www.ClinicalTrials.gov