Clinical Trials: Terms to Know
Clinical trial: multi-step process taking scientific research discoveries and findings from the laboratory (basic research) and applying them into real-world clinical settings (clinical research).
Informed or treatment consent form: document that outlines a patient’s role in receiving an experimental treatment, and the possible implications of proceeding with the investigational or unapproved treatment. It should provide a clear and detailed description of the treatment or procedure in plain language and explain a patient’s options for treatment, their rights and responsibilities, and the risks.
Investigational treatment: term sometimes used to describe stem cell treatments that are being tested in clinical trials but are not approved as effective and safe. Until formal approval, a stem cell treatment that is being tested in clinical trials is considered investigational. It is important to understand that testing a stem cell treatment in clinical trials does not guarantee that it will become an approved and established therapy.
Efficacy: the ability to produce a desired or intended result. Also called effectiveness.
Control group: Group of participants who do not receive the experimental treatment under investigation. Instead, they are given an existing approved treatment, or a placebo. This group is essential to determine the overall efficacy and safety of the experimental treatment under study.
Randomized clinical trial: participants are randomly assigned to receive different interventions in order to reduce bias.
Blinded clinical trial: participants do not know if they receive the treatment that is being assessed, a control treatment, or a placebo.
Placebo: an inactive treatment that is used as a comparison to help researchers determine whether a treatment is more effective than no treatment at all.
Phase 1: researchers test an experimental drug or treatment in a small group of people (20-100) for the first time. The researchers evaluate the treatment’s safety, determine a safe dosage range, and identify side effects.
Phase 2: the experimental drug or treatment is given to a larger group of people (up to 300) to see if it is effective and to further evaluate its safety.
Phase 3: the experimental study drug or treatment is given to large groups of people (300-3,000). Researchers confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.
Phase 4: post-marketing studies, which are conducted after a treatment is approved for use by the FDA, provide additional information including the treatment or drug’s risks, benefits, and best use.
